#urea cycle disorders key companies

[delveinsight12]

Urea Cycle Disorders Market Growth Anticipated by 2032 | Major Players: Aeglea BioTherapeutics, Immedica Pharma, Ultragenyx Pharmaceutical, Arcturus Therapeutics, expected to boost the market

In the market landscape of Urea Cycle Disorders, an impressive surge is expected during the study period spanning 2019 to 2032, according to the latest report titled  “Urea Cycle Disorders Market Insights, Epidemiology and Market Forecast 2032” from DelveInsight. 

The Urea Cycle Disorders market report sheds light on Urea Cycle Disorders current treatment practices, upcoming drugs in the Urea Cycle Disorders pipeline, market shares of individual therapies, and the anticipated trajectory of the Urea Cycle Disorders market size from 2019 to 2032 across the 7MM (the United States, the EU-4 comprising Italy, Spain, France, and Germany, the United Kingdom, and Japan).

Driving Forces Behind the Urea Cycle Disorders Market Growth

The market size shall grow during the forecast period, i.e., 2023–2032 owing to the increasing prevalence of Urea Cycle Disorders. The market size is expected to increase during the study period.

Discover the Anticipated Evolution and Growth of the Market @ Urea Cycle Disorders Therapeutics Market Forecast

Therapeutic Advancements and Emerging Treatments:

Urea Cycle Disorders Clinical Trial Progression: The market is set to experience significant growth, driven by the progression of emerging therapies expected for launch between 2023 and 2032. Pioneering companies, including Aeglea BioTherapeutics, Immedica Pharma, Ultragenyx Pharmaceutical, Arcturus Therapeutics, and others, are actively engaged in developing novel drugs for potential market entry.

Urea Cycle Disorders Innovative Therapies: Ongoing research and development activities are fostering the introduction of innovative therapies designed to address the signs and symptoms of Urea Cycle Disorders. Therapies such as LOARGYS (pegzilarginase), DTX301, LUNAR-OTC (ARCT-810), and others are driving the Urea Cycle Disorders market.

Urea Cycle Disorders Treatment Market

The treatment of disorders related to the urea cycle is a lifelong process aimed at managing symptoms and doesn't cure the disorder. Strategies include monitoring ammonia levels using blood tests (serum and plasma levels) at regular intervals and using the results to optimize treatment methods. In the current market scenario, recommended therapies to possibly slow the progression of the disease include sodium benzoate, sodium phenylacetate, Glycerol phenylbutyrate, sodium phenylbutyrate, and antiemetic agents. 

These drugs lower blood ammonia concentrations by conjugation reactions involving acylation of amino acids. For neonates with hyperammonemia, the immediate treatment goal is a rapid lowering of ammonia and reducing dietary protein intake. Hemodialysis is very effective at reducing plasma ammonia and should immediately be initiated if elevated hyperammonemia is observed. Ammonia scavenger medications such as AMMONUL IV are also useful. AMMONUL IV acts by removing glycine and glutamate from plasma thereby their reducing contribution to ammonia formation.

Urea Cycle Disorders Treatment Market

The dynamics of the Urea Cycle Disorders market are expected to change owing to a robust pipeline products. Some of the key players involved in the development of novel drugs are Aeglea BioTherapeutics, Immedica Pharma, Ultragenyx Pharmaceutical, Arcturus Therapeutics, and others.

LOARGYS (pegzilarginase) is an engineered human arginase I enzyme designed to degrade the amino acid arginine. Pegzilarginase is being developed to treat two extremes of arginine metabolism, including arginine excess in patients with Arginase 1 Deficiency, as well as some cancers which have been shown to have a metabolic dependency on arginine. The company has completed a Phase III clinical study which demonstrated reduction in blood arginine levels in patients with ARG1-D. The therapy was well tolerated among the patients. Recently, in October 2023, Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency has adopted a positive opinion recommending marketing authorization of LOARGYS (pegzilarginase) for the treatment of arginase 1 deficiency (ARG1-D) in patients two years and older. 

DTX301 is an investigational gene therapy being developed for the treatment of individuals with ornithine transcarbamylase (OTC) deficiency which is the most common urea cycle disorder. DTX301 is designed by Ultragenyx Pharmaceutical to deliver OTC gene expression in a durable fashion, with the goal of preventing or reducing the occurrence of complications associated with OTC deficiency. It has been granted Orphan Drug Designation in the United States, EU and United Kingdom and Fast Track Designation in the United States. Long term Phase I/II data demonstrated an acceptable safety profile and durable metabolic control and sustained responses. The company is conducting a Phase III (NCT05345171) trial to evaluate the efficacy of DTX301 on the improvement of ornithine transcarbamylase (OTC) function by maintaining safe plasma ammonia levels with removal of dietary protein restriction and alternative pathway medication.

LUNAR-OTC (ARCT-810) is messenger RNA (mRNA) coding for Ornithine Transcarbamylase (OTC) formulated in a lipid nanoparticle (LNP). Arcturus Therapeutics is developing mRNA medicines that enable OTC patients to make healthy functional OTC enzyme in the liver cells. The early phase studies demonstrated efficacy of the therapy in restoration of the urea cycle function to normal levels and increased survival. Currently, ARCT-810 is being investigated in Phase II (NCT05526066) clinical development to evaluate the safety, tolerability and pharmacokinetics of ARCT-810 in adolescent and adult participants with ornithine transcarbamylase deficiency.

Leading Urea Cycle Disorders Companies and Emerging Drugs: Pioneering companies such as Aeglea BioTherapeutics, Immedica Pharma, Ultragenyx Pharmaceutical, Arcturus Therapeutics, among others, are actively developing novel drugs for potential entry into the Urea Cycle Disorders market.

Urea Cycle Disorders Therapeutic Landscape: Key therapies identified for Urea Cycle Disorders treatment include LOARGYS (pegzilarginase), DTX301, LUNAR-OTC (ARCT-810), and more.

Urea Cycle Disorders Overview:

Urea cycle disorders (UCDs) are a group of rare genetic disorders that affect the body's ability to eliminate ammonia, a waste product formed during the breakdown of proteins. Normally, ammonia is converted into urea in the liver through a series of biochemical reactions known as the urea cycle. Urea is then excreted from the body through urine.

In individuals with urea cycle disorders, one of the enzymes involved in the urea cycle is deficient or dysfunctional, leading to the accumulation of ammonia in the blood. This elevated ammonia, known as hyperammonemia, can be toxic to the brain and nervous system, causing neurological damage and potentially life-threatening complications.

There are several types of urea cycle disorders, each caused by mutations in specific genes that encode enzymes critical to the urea cycle. These disorders include:

Ornithine Transcarbamylase (OTC) Deficiency: This is the most common urea cycle disorder, caused by a deficiency in the OTC enzyme. It is an X-linked disorder, primarily affecting males. Symptoms can range from mild to severe and may appear shortly after birth or later in life.

Carbamoyl Phosphate Synthetase (CPS) Deficiency: CPS deficiency is a rare autosomal recessive disorder caused by mutations in the CPS1 gene, leading to reduced or absent activity of the CPS enzyme. Symptoms can be severe and appear soon after birth.

Argininosuccinic Acid Synthetase (AS) Deficiency: AS deficiency results from mutations in the ASS1 gene, leading to decreased production of the AS enzyme. Symptoms can range from mild to severe and may present at different ages, from infancy to adulthood.

Arginase Deficiency: This disorder is caused by mutations in the ARG1 gene, resulting in a deficiency of the arginase enzyme. Symptoms often appear in early childhood and can vary in severity.

Symptoms of urea cycle disorders can include vomiting, lethargy, irritability, seizures, developmental delay, coma, and even death if not promptly treated. Diagnosis involves blood tests to measure ammonia levels and specific metabolites related to the urea cycle, as well as genetic testing to identify the specific enzyme deficiency.

Treatment aims to reduce ammonia levels and prevent its accumulation. This often involves dietary restrictions, medications like ammonia-scavenging drugs, and in severe cases, dialysis to remove excess ammonia from the blood. Long-term management by metabolic specialists and genetic counselors is essential for individuals with urea cycle disorders. Early detection and intervention are critical to minimize the risk of neurological damage and improve outcomes for affected individuals.

Key Facts Urea Cycle Disorders Market Report:

Key players such as Aeglea BioTherapeutics, Immedica Pharma, Ultragenyx Pharmaceutical, Arcturus Therapeutics, and others are investigating its candidates for Urea Cycle Disorders.

Urea Cycle Disorders pipeline includes the major therapies such as LOARGYS (pegzilarginase), DTX301, LUNAR-OTC (ARCT-810), and others. 

In December 2022, the US FDA approved OLPRUVA (sodium phenylbutyrate) to treat certain patients living with urea cycle disorders (UCDs). Acer Therapeutics presented data from a survey designed to quantify preferences of healthcare providers for Urea Cycle Disorders (UCDs) at the 44th Annual Meeting of the Society for Inherited Metabolic Disorders (SIMD) on March 18th to 21st.

Urea Cycle Disorders Epidemiology Segmentation:

According to NORD, OTC deficiency affects males more often than females and most males are symptomatic in nature. In males, symptoms typically begin during the first few days of life.

According to reports published by the Centers for Disease Control and Prevention (2022), the anticipated incidence of UCD is 1 in 8,500 births. The research findings suggest that many of the cases of UCD persist undiagnosed. In some of the cases, the newborn with UCD dies without a conclusive diagnosis.

According to the American Association for Clinical Chemistry (2019), the incidence of Urea Cycle Disorders, or UCDs, in the US is estimated to be around 1 in 8,200 births. The mortality rate is 24% in neonatal cases, and 11% in later onset cases.

For in-depth insights, access the full report @ Urea Cycle Disorders Market Outlook 2032

[thesunshinereporter]

Urea Cycle Disorder Market is Expected to Expand at a Healthy Growth Rate During the Study Period (2019 -2032)|Key Companies – Acer Therapeutics, Poseide Therapeutics, Kaleido Biosciences

http://dlvr.it/Sw64mn

[digiresearcher]

Sodium Phenylbutyrate Market: Rising Demand for Treatment of Genetic Disorders Drives Growth

Market Overview: The global Sodium Phenylbutyrate Market is estimated to be valued at US$1.92 billion in 2023 and is expected to exhibit a CAGR of 6.7% over the forecast period from 2023 to 2030, according to a new report published by Coherent Market Insights. Sodium Phenylbutyrate is a medication used for the treatment of genetic disorders, such as urea cycle disorders and certain types of cancer. It is known for its ability to reduce ammonia levels in the body and has been proven to be effective in managing various diseases. The rise in the prevalence of genetic disorders and the need for effective treatment options are driving the demand for sodium phenylbutyrate. Market Key Trends: One key trend in the Sodium Phenylbutyrate market is the increasing focus on research and development activities. Pharmaceutical companies and research institutions are investing in the development of innovative formulations and dosage forms of sodium phenylbutyrate to enhance its therapeutic efficacy. For example, Horizon Therapeutics plc has recently introduced a delayed-release tablet formulation of sodium phenylbutyrate, which offers improved patient compliance and convenience. Porter's Analysis: - Threat of new entrants: The high cost of research and development, along with stringent regulatory requirements, acts as a barrier to entry for new players in the sodium phenylbutyrate market. - Bargaining power of buyers: The limited availability of sodium phenylbutyrate and its crucial role in managing genetic disorders gives buyers less bargaining power. - Bargaining power of suppliers: The suppliers of sodium phenylbutyrate hold significant bargaining power due to their control over the production and distribution channels. - Threat of new substitutes: Currently, there are limited substitutes available for sodium phenylbutyrate in the treatment of genetic disorders. - Competitive rivalry: The sodium phenylbutyrate market is highly competitive, with the presence of key players such as Horizon Therapeutics plc, Ammonett Pharma LLC, and Synageva BioPharma Corp. These companies are focusing on product innovation and strategic collaborations to gain a competitive edge. Key Takeaways: - The global Sodium Phenylbutyrate Market is expected to witness high growth, exhibiting a CAGR of 6.7% over the forecast period, driven by the increasing prevalence of genetic disorders and the need for effective treatment options. - The Asia Pacific region is anticipated to be the fastest-growing and dominating region in the sodium phenylbutyrate market. The region's rising population, increasing healthcare expenditure, and growing awareness about genetic disorders contribute to market growth. - Key players operating in the global Sodium Phenylbutyrate market include Horizon Therapeutics plc, Ammonett Pharma LLC, Medison Pharma Ltd., Modern Dietetics Inc., Synageva BioPharma Corp., Swedish Orphan Biovitrum AB (Sobi), Entera Health, Inc., Dipharma Francis S.r.l., Ucyclyd Pharma, Inc. These companies are focusing on strategic collaborations, product launches, and research and development activities to expand their market presence and cater to the growing demand for sodium phenylbutyrate. In conclusion, the Sodium Phenylbutyrate market is experiencing significant growth due to the increasing prevalence of genetic disorders and the need for effective treatment options. The market's key trends include a focus on research and development activities, while Porter's analysis highlights the competitive landscape and factors influencing the market dynamics. With the Asia Pacific region expected to witness rapid growth and key players striving for innovation and market expansion, the future of the Sodium Phenylbutyrate market looks promising.

[cmipooja]

U.S. Urea Cycle Disorders Treatment Market Is Estimated To Witness High Growth Owing To Increasing Focus on Rare Diseases

The U.S. Urea Cycle Disorders (UCD) Treatment Market is estimated to be valued at US$ 510.0 million in 2023 and is expected to exhibit a CAGR of 3.56% over the forecast period 2023-2030, as highlighted in a new report published by Coherent Market Insights. Market Overview: Urea Cycle Disorders (UCD) are a group of rare genetic disorders that affect the body's ability to metabolize ammonia, resulting in its accumulation in the blood. This can lead to serious health complications and even death if left untreated. UCD treatment aims to manage the levels of ammonia in the body through medication and dietary modifications. The U.S. Urea Cycle Disorders Treatment Market offers a wide range of products and therapies to effectively manage UCD. These include oral medications, protein substitutes, and medical foods. These products help in reducing ammonia levels and improving overall health outcomes for patients with UCD. The market is driven by the increasing prevalence of UCD, growing awareness about rare diseases, and advancements in treatment options. Market Key Trends: One key trend in the U.S. Urea Cycle Disorders Treatment Market is the increasing focus on rare diseases. Rare diseases, including UCD, are gaining more attention from healthcare providers, pharmaceutical companies, and regulatory authorities. This is due to the significant unmet medical needs of patients with rare diseases and the potential for innovative treatments. For example, Horizon Therapeutics Plc and Bausch Health Companies Inc have developed medication for UCD that has shown promising results in clinical trials. These innovative therapies have the potential to revolutionize the treatment landscape for UCD and improve the quality of life for patients. PEST Analysis: Political: The political landscape plays a crucial role in the availability and accessibility of UCD treatment options. Government policies and regulations impact drug development, pricing, and reimbursement, which can have a significant impact on the U.S. Urea Cycle Disorders Treatment Market. Economic: The economic factors influencing the U.S. Urea Cycle Disorders Treatment Market include healthcare spending, insurance coverage, and affordability of treatments. The availability of healthcare resources and funding for research and development also impact the market. Social: The social factors affecting the U.S. Urea Cycle Disorders Treatment Market include the prevalence of UCD, awareness about rare diseases, and patient advocacy groups. The increasing emphasis on personalized medicine and patient-centric care also influences the market. Technological: Advancements in technology, such as genetic testing and precision medicine, have opened new avenues for the diagnosis and treatment of UCD. These technologies enable early detection, personalized treatment plans, and targeted therapies for patients. Key Takeaways: 1: The U.S. Urea Cycle Disorders Treatment Market Demand is expected to witness high growth, exhibiting a CAGR of 3.56% over the forecast period. This growth is driven by increasing awareness about rare diseases and advancements in treatment options for UCD. For example, innovative therapies developed by Horizon Therapeutics Plc and Bausch Health Companies Inc have shown promising results in clinical trials. 2: The United States is expected to be the fastest-growing and dominating region in the U.S. Urea Cycle Disorders Treatment Market. The high prevalence of UCD in the country, robust healthcare infrastructure, and favorable regulatory environment contribute to its market dominance.

[healthpulsetoday]

Argininosuccinic Aciduria: Moving Towards Effective Treatment Strategies

Argininosuccinic aciduria (ASA) is a rare genetic disorder caused by the deficiency of argininosuccinate lyase, an enzyme involved in the urea cycle. The urea cycle is responsible for removing ammonia from the body, and without the enzyme, ammonia accumulates in the blood, causing neurological and developmental problems. Argininosuccinic aciduria is a severe condition that can affect infants and children, and in some cases, adults. Currently, there is no treatment for Argininosuccinic Aciduria, and the cure is mainly focused on managing the symptoms and reducing the levels of ammonia in the blood.

Argininosuccinic aciduria is a rare condition, and its prevalence is estimated to be one in 70,000 to 218,000 individuals worldwide. The symptoms of Argininosuccinic aciduria can vary from person to person, but some common signs include vomiting, seizures, developmental delays, intellectual disability, and behavioral problems. In some cases, people with Argininosuccinic aciduria may have brittle hair or hair loss, which can be a noticeable sign of the condition. The life expectancy of individuals with Argininosuccinic aciduria can vary depending on the severity of the condition, but early diagnosis and treatment can improve the outcomes and increase the lifespan.

According to the report by Delveinsight Business Research, Argininosuccinic aciduria market is expected to grow significantly in the coming years due to the increasing prevalence of the disease and the launch of new treatments. The report provides a comprehensive analysis of the market trends, drivers, challenges, and opportunities, along with the competitive landscape and pipeline analysis of the key players in the market.

Currently, the treatment options for Argininosuccinic aciduria are limited, and the focus is on managing the symptoms and reducing the levels of ammonia in the blood. This includes a low-protein diet, medications to remove excess ammonia, and supplements to support the urea cycle. However, these treatments are not always effective, and there is a need for more innovative and targeted therapies.

Researchers and pharmaceutical companies are working to develop new treatments for Argininosuccinic aciduria, and there are several promising candidates in the pipeline. One approach is to develop gene therapies that can correct the genetic defect that causes Argininosuccinic aciduria. Another approach is to develop enzyme replacement therapies that can supplement the missing or deficient enzyme. These therapies are still in the early stages of development, but they hold promise for improving the outcomes for individuals with Argininosuccinic aciduria.

The report by Delveinsight Business Research highlights the pipeline analysis of the key players in the Argininosuccinic aciduria market, including their product portfolio, development stages, and clinical trials. The report also offers insights into the regulatory landscape, reimbursement policies, and pricing analysis for the Argininosuccinic aciduria market.

In conclusion, Argininosuccinic aciduria is a rare genetic disorder that can have severe neurological and developmental effects. There is currently no cure for Argininosuccinic aciduria, and the treatment options are limited. However, with the ongoing research and development of new therapies, there is hope for the future. Innovative treatments, such as gene therapies and enzyme replacement therapies, are expected to launch in the next decade, which could significantly improve the outcomes for individuals with Argininosuccinic aciduria. It is essential to continue supporting research efforts and raising awareness of this rare condition to improve the diagnosis, treatment, and management of Argininosuccinic aciduria.

Asset Priortization in Healthcare

R&D Analysis in Healthcare

[kritikarehani]

Key Pharma Players driving the Urea Cycle Disorders Market

Pharma companies like Ultragenyx Pharmaceutical, Acer Therapeutics, Aeglea BioTherapeutics, Promethera Biosciences and Kaleido Biosciencesare expected to create a positive shift in the Urea Cycle Disorders Market in upcoming years.

Source: https://www.delveinsight.com/report-store/urea-cycle-disorders-market

[healthcare-market]

Sodium Phenylbutyrate Drugs Market Estimated to Expand at a Robust CAGR by 2025

Sodium Phenylbutyrate Drugs Market: Introduction

Sodium phenylbutyrate drugs are prescribed for adjunctive therapy in the chronic management of urea cycle disorders. They are primarily administered orally, in equally divided doses, with each meal or feeding. Divalproex (Depakote), haloperidol (Haldol), valproic acid (Depakene, Stavzor) may interact with sodium phenylbutyrate, if taken simultaneously. Buphenyl is a brand name of sodium phenylbutyrate, approved by the FDA.

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Key Drivers and Restraints of Global Sodium Phenylbutyrate Drugs Market

· Urea cycle disorders (UCDs) are inborn errors of metabolism (IEMs) resulting from defects in any 1 of the six enzymes or 2 transporters involved in the hepatic removal of ammonia from the bloodstream by conversion to urea, which is excreted by the kidneys. UCDs include carbamoyl phosphate synthase (CPS) deficiency, ornithine transcarbamylase (OTC) deficiency, argininosuccinate synthetase deficiency (citrullinemia), argininosuccinate lyase deficiency (argininosuccinic aciduria), and arginase deficiency (argininemia). Overall incidence of UCD, globally, varies from 1:8000 to 1:44,000 births. An estimated cumulative incidence of UCDs in Europe is approximately 1 in 51,946. Sodium phenylbutyrate drugs are utilized for the treatment of urea cycle disorders. Thus, an increase in cases of UCDs fuel the market.

· Increased research & development initiatives undertaken by research & academic institutes supported by pharmaceutical companies as well as governments are estimated to drive sodium phenylbutyrate drugs market

· Side effects related to the intake of sodium phenylbutyrate drugs may hamper the market. Common side effects may include missed menstrual periods or changes in the regular cycle, loss of appetite, headache, nausea or vomiting, lower back, side, or stomach pain.

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Oral Segment Witness Highest Growth

· Based on route of administration, the global sodium phenylbutyrate drugs market can be divided into oral and nasogastric intubation

· The oral segment accounted for a prominent share of the market in 2019. Sodium phenylbutyrate is available in tablet or powder form. Nasogastric intubation involves the insertion of a plastic tube through the nose, past the throat, and down into the stomach. Nasogastric tube (NGT) insertion is done through anesthetized procedure for critically ill patients who cannot swallow a tablet through oral intake.

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Hospital Pharmacies to Dominate Global Sodium Phenylbutyrate Drugs Market

· Based on distribution channel, the global sodium phenylbutyrate drugs market can be divided into: hospital pharmacies, online and retail pharmacies

· In terms of revenue, the hospital pharmacies segment accounted for a notable share of the global market in 2019, followed by the retail pharmacies segment. Within a hospital, the hospital pharmacy dispenses inpatient medications. Hospital pharmacies usually stock a larger range of medications. Expansion of hospital pharmacies is attributed to offering quality medicines to patient by purchasing directly from drug manufacturers, thus preventing the issues of counterfeit medicines.

· The online segment is estimated to expand at a significant CAGR during the forecast period. Increasing penetration of internet, worldwide, rising investments made by private companies, and an increase in the number of steps taken by government bodies are major factors propelling the segment

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North America to Dominate Global Sodium Phenylbutyrate Drugs Market

· In terms of region, the global sodium phenylbutyrate drugs market can be divided into: North America, Europe, Asia Pacific, Latin America, and Middle East & Africa

· In terms of revenue, North America dominated the sodium phenylbutyrate drugs market. Increase in incidence of urea cycle disorders, rise in number of FDA approvals, and continuous merger & acquisition between companies contribute to the leading share held by the region. According to data published in NCBI in 2013, incidence of urea cycle disorders is at 1 in 35,000 live births in the U.S. or about 113 new patients per year.

· The sodium phenylbutyrate drugs market in Asia Pacific is anticipated to be driven by an increase in the number of target patients, rise in healthcare expenditure, and investments by various companies in research & development activities in the region.

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[thesunshinereporter]

Urea Cycle Disorder Market is Expected to Expand at a Healthy Growth Rate During the Study Period (2019 -2032)|Key Companies – Acer Therapeutics, Poseide Therapeutics, Kaleido Biosciences

http://dlvr.it/Sw64jx

[freesuitwhispers]

Global Urea Cycle Disorder (UCD)   Market - Growth Drivers, Opportunities And Forecast Analysis To 2020-2026

Summary - A new market study, “ Global Urea Cycle Disorder (UCD)   Market - Growth Drivers, Opportunities And Forecast Analysis To 2020-2026 ” has been featured on WiseGuyReports.  

Urea cycle disorders (UCDs) are a group of orphan inherited defects of six enzymes and two transporters that constitute the urea cycle in the periportal liver cells. The age of onset symptoms vary between the different types of UCDs. UCDs often occurs during the period of infancy due to triggered postnatal catabolism. The diagnosis is made by analysis of the urine and blood for abnormal metabolites (substance produced by metabolism), and high ammonia levels. Frequent blood tests are done to monitor ammonia levels. The treatment of urea cycle disorders consists of dietary management to limit ammonia production. A liver transplant can be an effective treatment for urea cycle disorder for the proper functioning of urea cycle.

 The urea cycle disorder treatment market in the U.S. and Europe is expected to grow rapidly in the next few years. Several pharmaceutical companies are under the development process of their late stage pipeline drugs for urea cycle disorders. Factors such as, increasing healthcare expenditures in the U.S, rise in the U.S. pharmaceutical R&D expenditures and collaboration between pharmaceutical companies and research institutes have driven the growth of the U.S. urea cycle disorder treatment market. Further, upcoming launches of anti-UCD drugs would contribute to the expansion of the urea cycle disorder treatment market in the U.S. as well as in Europe.

Also Read : http://heraldkeeper.com/news/urea-cycle-disorder-ucd-market-global-share-size-trends-and-growth-analysis-forecast-to-2020-2024-513653.html

 The U.S. urea cycle disorder treatment market is dominated by certain key players including Horizon Pharma Inc., Ultragenyx Pharmaceuticals Inc., Aeglea Bio Therapeutics and Synlogix. Among all the key players, Horizon Pharma ruled the U.S. UCD treatment market owing to the strong performance of its product. Horizon’ Ravicti is the market leader due its strong curative impact on the patients with UCD. The remaining key players have their products under the development process of pipeline drugs for UCDs. There would be a generic competition between the upcoming anti-UCD drugs in the coming years.

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[market-insights-dbmr]

Global Maple Syrup Urine Disease Treatment Market Analysis, Opportunities, Innovations with Economic Conditions By 2026

Global maple syrup urine disease treatment market is expected to grow at a substantial CAGR in the forecast period of 2019-2026. Emergence of drugs used to treat risk associated with maple syrup urine disease and high demand of disease specific novel therapies are the key factors that fueling the market growth.

Few of the major competitors currently working in the global maple syrup urine disease treatment market are CENTOGENE AG, Acer Therapeutics Inc, HemoShear Therapeutics, LLC, Recordati SpA, Jo Mar Laboratories, Siddhi Vinayaka Spechem Private Limited, Baxter, EXOCOBIO, Pfizer Inc, and others.

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Key Developments in the Market:

In November 2018, Recordati SpA received an Orphan Drug designation from the European Commission for REC 0545, a serotonin 1A receptor antagonist for the treatment of maple syrup urine disease. With this designation company enables to obtain ten years marketing exclusivity upon approval of the product in the designated indication.

In May 2016, Acer Therapeutics Inc secured fund of USD 8.15 million through Series B Financing round for developing first-in-class ACER-002 for the treatment of vascular Ehlers-Danlos Syndrome (vEDS) as well as Maple Syrup Urine Disease (MSUD) and Urea Cycle Disorder (UCD). This funding aims to speed the development of an exciting new class of therapeutics to treat patients suffering from inherited metabolic disorders.

Competitive Analysis:

Global maple syrup urine disease treatment market is highly fragmented and the major players have used various strategies such as new product launches, expansions, agreements, joint ventures, partnerships, acquisitions, and others to increase their footprints in this market. The report includes market shares global maple syrup urine disease treatment market for Global, Europe, North America, Asia-Pacific, South America and Middle East & Africa.

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Segmentation: Global Maple Syrup Urine Disease Treatment Market

Global Maple Syrup Urine Disease Treatment Market By Type (Classic MSUD, Intermediate MSUD, Intermittent MSUD and Others), Treatment Type (Medication, Surgery), Drugs (High-Calorie BCAA-Free Amino Acids, Caloric Supplementation and Others), Route of Administration (Oral, Injectable), End- Users (Hospitals, Homecare, Specialty Clinics, Others), Geography (North America, South America, Europe, Asia-Pacific, Middle East and Africa) - Industry Trends  and Forecast to 2026

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[kritikarehani]

Urea Cycle Disorders Market Scenario

A Urea Cycle Disorder (UCD) is a genetic disorder that results in a deficiency of some major enzymes in the urea cycle. These enzymes are responsible for removing ammonia from the blood stream. The urea cycle involves a series of biochemical steps in which nitrogen, a waste product of protein metabolism, is changed to a compound called urea and removed from the blood.

Normally, the urea is removed from the body through the urine. In urea cycle disorders, nitrogen builds up in the blood in the form of ammonia, a highly toxic substance, resulting in hyperammonemia (elevated blood ammonia). Ammonia then reaches the brain through the blood, where it can cause irreversible brain damage, coma, and/or death. The onset and severity of urea cycle disorders is highly variable. The severity correlates with the amount of urea cycle enzyme function.

DelveInsight's "Urea Cycle Disorders - Market Insights, Epidemiology, and Market Forecast-2030" report delivers an in-depth understanding of the Urea Cycle Disorders, historical and forecasted epidemiology as well as the Urea Cycle Disorders market trends in the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom) and Japan.

Geography Covered

· The United States

· EU5 (Germany, France, Italy, Spain, and the United Kingdom)

· Japan

Study Period: 2017-2030

Urea Cycle Disorders Disease Understanding and Treatment Algorithm

A few of the enzymes which are involved in the Urea Cycle Disorder, collectively known as inborn errors of urea synthesis, or urea cycle enzyme defects; each is referred to by the initials of the missing enzyme: CPS1 (Carbamoyl Phosphate Synthetase), NAGS (N-Acetylglutamate Synthetase), OTC Deficiency (Ornithine Transcarbamylase), AS (Argininosuccinic Acid Synthetase (Citrullinemia)), ASL (Argininosuccinate Lyase (Argininosuccinic Aciduria)), and AG (Arginase).

In children with severe UCD, the symptoms will develop within the first 24 hours of life. Symptoms in children with mild or moderate UCD, who do not show symptoms until early childhood, may include: Vomiting, nausea, Mental confusion or hyperactive behavior, Tired often and / or hard to awaken, Coma, etc.

Treatment is a lifelong process that doesn't cure the condition, but it can effectively manage the symptoms. The child's treatment probably will involve:

•       Low protein, high-calorie diet

•       Medications

•       Amino acid supplements

•       Liver transplantation

The DelveInsight Urea Cycle Disorders market report gives a thorough understanding of the Urea Cycle Disorders by including details such as disease definition, symptoms, causes, pathophysiology, diagnosis and treatment. 

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Urea Cycle Disorders Epidemiology  

Urea cycle disorders are autosomal recessive disorders with the exception of ornithine transcarbamylase deficiency, which is X-linked. Neonates with absent urea cycle enzyme activity typically present with hyperammonemic coma within the first week after birth. Outcomes can be severe, with high morbidity and mortality approaching 50% (Batshaw and Monahan, 1987). This disorder is one of the most common inborn errors of metabolism in the liver, with an estimated Incidence ranging from 1:30,000 to 1:46,000 live births. Here are some of the epidemiological studies.

The Urea Cycle Disorders epidemiology division provide insights about historical and current Urea Cycle Disorders patient pool and forecasted trend for every seven major countries. It helps to recognize the causes of current and forecasted trends by exploring numerous studies and views of key opinion leaders. This part of the DelveInsight report also provides the diagnosed patient pool and their trends along with assumptions undertaken.  

Urea Cycle Disorders Drug Chapters

The dynamics of Urea Cycle Disorders market is anticipated to change in the coming years owing to the improvement in the diagnosis methodologies, raising awareness of the diseases, incremental healthcare spending across the world, and the expected launch of emerging therapies during the forecast period of 2020–2030.    

Drug chapter segment of the Urea Cycle Disorders report encloses the detailed analysis of Urea Cycle Disorders marketed drugs and late stage (Phase-III and Phase-II) pipeline drugs. It also helps to understand the Urea Cycle Disorders clinical trial details, expressive pharmacological action, agreements and collaborations, approval and patent details, advantages and disadvantages of each included drug and the latest news and press releases.

Marketed Drugs 

The report provides the details of the marketed product available for Urea Cycle Disorders treatment. 

Urea Cycle Disorders Emerging Drugs 

The report provides the details of the emerging therapies under the late and mid-stage of development for Urea Cycle Disorders treatment. 

Urea Cycle Disorders Market Outlook

Recent advances in the treatment of inborn errors of urea synthesis have significantly decreased mortality. The treatment of urea cycle disorders consists of dietary management to limit ammonia production, in conjunction with medications and/or supplements which provide alternative pathways for the removal of ammonia from the bloodstream. The aim is to correct biochemical abnormalities and ensure adequate nutritional intake. Treatment involves compounds that increase the removal of nitrogen waste. These compounds convert nitrogen into products other than urea, which are then excreted; hence, the load on the urea cycle is reduced. The first compounds to be used were sodium benzoate and arginine. Later, phenylacetate was used, which has now been replaced by phenylbutyrate.

Hemodialysis is very effective at reducing plasma ammonia and should immediately be initiated if elevated hyperammonemia is observed. Ammonia scavenger medications such as Ammonul IV are also useful. Ammonul IV acts by removing glycine and glutamate from plasma thereby their reducing contribution to ammonia formation.

The Urea Cycle Disorders market outlook of the report helps to build the detailed comprehension of the historic, current, and forecasted Urea Cycle Disorders market trends by analyzing the impact of current therapies on the market, unmet needs, drivers and barriers and demand of better technology.

This segment gives a thorough detail of Urea Cycle Disorders market trend of each marketed drug and late-stage pipeline therapy by evaluating their impact based on annual cost of therapy, inclusion and exclusion criteria's, mechanism of action, compliance rate, growing need of the market, increasing patient pool, covered patient segment, expected launch year, competition with other therapies, brand value, their impact on the market and view of the key opinion leaders. The calculated market data are presented with relevant tables and graphs to give a clear view of the market at first sight. 

According to DelveInsight, Urea Cycle Disorders market in 7MM is expected to change in the study period 2017-2030.

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[healthcaretrends-blog]

Carglumic Acid Market Opportunities in Upcoming years 2019

Carglumic acid is an orphan drug used for the treatment of hyperammonemia. Hyperammonemia is a rare genetic disorder characterized by elevated level of ammonia in the blood. Increased entry of ammonia is a primary cause of neurological disorders such as congenital deficiencies of urea cycle enzymes, hepatic encephalopathy, Reye’s syndrome, several other metabolic disorders, and some toxic encephalopathy.

Global Carglumic Acid Market Drivers

Increasing research and development is expected to drive the global carglumic acid market growth. For instance, in April 2017, Dipharma S.A, a Swiss pharmaceutical company, developed fast dissolving formulation of carglumic acid tablets. These tablets are stable at room temperature and are used for the treatment and management of rare, life threatening inborn metabolic disorders affecting the urea cycle. Furthermore, increasing approvals is expected to boost the global carglumic acid market value. For instance, in September 2015, Orphan Europe announced that carbaglu (carglumic acid) is approved by the Health Canada, as an adjunctive therapy for the treatment and improvement of acute hyperammonemia or as maintenance therapy for chronic hyperammonemia.

Global Carglumic Acid Market Restraints

High cost of carglumic acid tablet is expected to hamper the global carglumic acid market growth. For instance, in April 2017, according to P&T Community report, Carbaglu (containing Carglumic acid) is an expensive prescription medicine in the world. This drug costs around US$ 1042.12 for 5 tablets. Furthermore, side effects associated with carglumic acid is expected to the hinder the global carglumic acid market growth. For instance, in May 2018, according to Drugs@FDA, carglumic acid can cause serious side effects such as ear infection, anemia, somnolence, pneumonia, influenza, hyperhidrosis, asthenia, anorexia.

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Global Carglumic Acid Market regional Analysis

North America is expected to hold dominant position in the carglumic acid market over the forecast period, owing to increasing collaborations by key players. For instance, in April 2017, Recordati Rare Diseases Canada, Inc. partnered with the Italy-based Recordati group, which is exclusive provider of several rare disease treatment including Carbaglu (Carglumic acid), Cystadane (Betaine anhydrous), and Cosmegen (Dactinomycin) in Canada. Furthermore, Asia Pacific is also projected to witness significant growth in the global carglumic acid market, owing to increasing initiatives by government to offer better healthcare facilities. For instance, in February 2019, China announced the collaborative network with various hospitals for the rare disease diagnosis and treatment. Under this network, 324 hospitals are selected according to their capacity and experience in treating patients with rare diseases. This collaboration will facilitate the timely transfer of difficult and complicated cases of rare diseases such as hyperammonemia between hospitals and provide the quality medical resources to them.

Key players operating in the global carglumic acid include Manus Aktteva Biopharma LLP., Civentichem LLC., Recoedati S.p.A, and Dipharma Francis S.r.I

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[coolluminaryland-blog1]

Neurometabolic Disorder Market Highly Favorable to the Growth Rate by 2014 to 2020

A Neurometabolic disorder is a group of individually rare and progressive disorders limited to symptomatic patient care. The analytical and clinical research result predicts significant development in explaining the molecular genetics, biochemical irregularities, pathogenesis of this disorder and diagnostic testing. The major symptoms of neurometabolic disorders are caused by progressive destruction of mental, motor and perceptual functions. These disorders also include seizures and loss of function associated with early death (before adulthood). Neurometabolic disorders primarily affect multiple organs and their symptoms vary from acute life-threatening disease to sub-acute progressive degenerative disorders.

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The most common neurometabolic disorders are amino acid apathies and organic acidurias followed by lipofuscinoses, congenital lactic acidosis, neuronal ceroid urea cycle disorders, peroxisomal disorders, while the less frequent are glycoprotein, mucopolysaccharidoses, degradation disorders, sphingolipidoses, and fatty acid oxidation disorders. The neurometabolic disorders market can be segmented on the basis of various disorders such as Pompe Disease, Fabry Disease, Gaucher’s Disease, Niemann-Pick Type C Disease and Mucopolysaccharidosis VI.Gaucher’s disease is a genetic disorder in which lipid (fatty substance) accumulates in the cells and certain organs. The disorder is differentiated by fatigue, low blood platelets, anemia, bruising, enlargement of liver and spleen. The major products used in treatment of Gaucher’s disease are Cerezyme manufactured by Genzyme (subsidiary of Sanofi SA) and VPRIV (velaglucerase alfa for injection) manufactured by Shire Plc. Pompe Disease is one of the rarest diseases, hereditary and often lethal disorder that disables the skeletal and heart muscles. It causes mutation in acid alpha-glucosidase (GAA) enzyme producing gene that tends to accumulate excess amounts of lysosomal glycogen mount up in various parts of the body, cardiac and skeletal muscles are the most fatally affected. The products used in treatment of Pompe Disease are Myozyme/Lumizyme manufactured by Genzyme (subsidiary of Sanofi SA). Fabry disease is a rare form of genetic lysosomal storage disease leading to deficiency in enzyme alpha galactosidase A (a-GAL A) that accounts for the associated clinical abnormalities of brain, eye, skin, heart, kidney and peripheral nervous system.

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The treatment regime of Fabry disease includes Fabrazyme that is manufactured by Genzyme (subsidiary of Sanofi SA) and Replagal manufactured by Shire Plc. Niemann-Pick Type C Disease is a lysosomal storage disease related with the mutations in NPC-1 and NPC-2 genes where the transport system related with cholesterol and glycolipids is disrupted and which leads to their excessive accumulation. The treatment for Niemann-Pick Type C includes Zavesca a product of Actelion Pharmaceuticals. 

Mucopolysaccharidosis VI consists of malfunctioned activity of the lysosomal enzymes, which interrupts the degradation of mucopolysaccharides which leads to abnormal buildup of keratan sulfate, dermatan sulfate, heparan sulfate. The standard treatment against mucopolysaccharidosis VI is with Naglazyme a product of BioMarin Pharmaceutical, Inc.The growth in institutional and research fields form transcriptomics, proteomics, metabolomics and lipidomics is expected to develop more advanced diagnostics and treatment. The various economic leverages provided by USFDA for orphan drug and diagnostics manufacturing companies are expected to grow the market. 

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Various institutional bodies investing the knowledge and economic sources in orphan disease research incur funding from various sources that is expected to contribute to sort out the role of very rarely studied organelle in neurodegenerative diseases and aging.Some of the major players in the Neurometabolic disorders market are Shire Plc, BioMarin Pharmaceutical, Inc., Actelion Pharmaceuticals Ltd., Sanofi SA, Pfizer, Inc., GlaxoSmithKline, Amicus Therapeutics, Inc. and others. The neurometabolic disorders market was dominated by Sanofi SA, GlaxoSmithKline, Amicu Therapeutics, Inc, and Pfizer, Inc.

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[sandlerresearch]

Urea Cycle Disorders Global Clinical Trials Review, H2, 2018 published on

http://www.sandlerresearch.org/urea-cycle-disorders-global-clinical-trials-review-h2-2018.html

Urea Cycle Disorders Global Clinical Trials Review, H2, 2018

Urea Cycle Disorders Global Clinical Trials Review, H2, 2018

Summary

GlobalData’s clinical trial report, “Urea Cycle Disorders Global Clinical Trials Review, H2, 2018” provides an overview of Urea Cycle Disorders clinical trials scenario. This report provides top line data relating to the clinical trials on Urea Cycle Disorders. Report includes an overview of trial numbers and their average enrollment in top countries conducted across the globe. The report offers coverage of disease clinical trials by region, country (G7 & E7), phase, trial status, end points status and sponsor type. Report also provides prominent drugs for in-progress trials (based on number of ongoing trials). GlobalData Clinical Trial Reports are generated using GlobalData’s proprietary database – Pharma eTrack Clinical trials database. Clinical trials are collated from 80+ different clinical trial registries, conferences, journals, news etc across the globe. Clinical trials database undergoes periodic update by dynamic process.

The report enhances the decision making capabilities and helps to create an effective counter strategies to gain competitive advantage.

*Note: Certain sections in the report may be removed or altered based on the availability and relevance of data for the indicated disease.

Scope

– The report provides a snapshot of the global clinical trials landscape – Report provides top level data related to the clinical trials by Region, Country (G7 & E7), Trial Status, Trial Phase, Sponsor Type and End point status – The report reviews top companies involved and enlists all trials (Trial title, Phase, and Status) pertaining to the company – The report provides all the unaccomplished trials (Terminated, Suspended and Withdrawn) with reason for unaccomplishment – The Report provides enrollment trends for the past five years – Report provides latest news for the past three months

*Note: Certain sections in the report may be removed or altered based on the availability and relevance of data for the indicated disease.

Reasons to buy

– Assists in formulating key business strategies with regards to investment – Helps in identifying prominent locations for conducting clinical trials which saves time and cost – Provides top level analysis of Global Clinical Trials Market which helps in identifying key business opportunities – Supports understanding of trials count and enrollment trends by country in global therapeutics market – Aids in interpreting the success rates of clinical trials by providing a comparative scenario of completed and uncompleted (terminated, suspended or withdrawn) trials – Facilitates clinical trial assessment of the indication on a global, regional and country level

*Note: Certain sections in the report may be removed or altered based on the availability and relevance of data for the indicated disease.

[repmywind02199]

SynBioBeta and the current biorevolution

SynBioBeta and the current biorevolution

Startups and individuals on the cusp of biological breakthroughs.

San Francisco is largely a collection of peninsulas and inlets, irregular land masses that singularly jut out into cold Pacific waters and isolate themselves by virtue of unavoidable geography. Travel to different parts of the bustling metropolis and you’ll witness an array of cultures and styles so distinctive they become complementary in their diversity alone; Mission District, Fisherman’s Wharf, Little Italy, Chinatown, each with individual aesthetics and atmosphere adding to the depiction of San Francisco as a microcosm for the world.

Unsurprisingly, the city’s tendency toward eclecticism doesn’t cease with street style. Industry, even at the highest levels of technological development, is pervaded by this intense desire for diversification coupled with community building. There is no better example of such initiative than SynBioBeta, an annual synthetic biology conference founded by Brown alumnus and NASA affiliate, John Cumbers. If San Francisco is a microcosm for the world, then surely SynBioBeta is the synthetic biology industry’s own miniature.

The 2017 San Francisco convention, housed within UCSF’s Mission Bay Conference Center, is an energetic forum for industry innovators and investors alike. The reception lobby is buzzing. Business-clad professionals mingle over coffee and work descriptions, all the while seeking opportunities for advancement of their companies’ roles in the network. The growing magnitude of businesses here is akin to adaptive radiation as one group—science-minded individuals—has rapidly speciated to fill several different industry niches. This diverse collection of producers is accurately described by CEO and co-founder of Twist Bioscience, Emily Leproust.

Leproust views the synthetic biology industry as an “ecosystem that will grow the pie,” with each company specializing in one area and driving efficiency in that way. There is an overarching attitude that it will take many companies, not one monopoly, to further development, and in this conception of the industry, Twist Bioscience, a leading manufacturer in the commercial synthesis of DNA, sees itself as a “facilitator for exponential growth.”

The company just announced its unprecedented one billion synthetic base pair deal with Ginkgo Bioworks that will support biological expansion into new industries. Twist’s production capabilities enable this type of large-scale manufacturing, producing something close to one-third of the world’s synthetic DNA, due to their main point of difference: a silicon platform. While DNA is typically synthesized on a 96-well plastic plate, Twist has developed a silicon alternative that, according to Leproust, “microscales 1 gene to 10,000 genes” and “gives you more scale while reducing your price per base.” This enables purchasers of synthetic DNA to receive their orders on a faster time scale and at a larger volume. Leproust believes this process impacts researchers in that “the more DNA you get, the faster you learn,” and in an industry where time is money, efficiency seems to be key.

With this type of technology becoming available, it isn’t difficult to imagine a world where synthetic biology enters the mainstream market. In fact, Leproust paints an intriguing picture of the market in which biology not only becomes a factor of production, but becomes a commonplace one.

“Cool things become the product. What enables the iPhone is hardcore physics, but what consumers have is a product,” says Leproust. She envisions a future in which “[consumers] forget about the biology that is behind the products” and instead simply enjoy the benefits of such goods or services.

Figure 1. Emily LeProust, CEO of Twist Bioscience. Image source: SynBioBeta, used with permission.

Twist Bioscience certainly isn’t the only believer in the future of synthetic biology. The numerous panels, presentations, and booths at SynBioBeta all revolve around the idea of a “biorevolution” and how producers can address global problems.

The main themes of the conference include biomaterials and consumer products, cell factories for biopharmaceuticals, and big data and artificial intelligence’s connection to biology. Each segment highlights pioneering individuals with specific visions for the future of synthetic biology and innovative companies working to produce high through-put in order to impact the state of today’s market.

Biomaterials and consumer products

The science of biomaterials is about 50 years old, an infant in the grand scale of scientific discoveries. Two companies featured at SynBioBeta are taking the concept of engineered substances for consumers and bringing it to the fashion industry.

Modern Meadow, a biofabrication company, poses the question of how to clothe the world’s growing population. Its answer comes in the form of synthetic leathers, an attempt to turn an animal product into a textile. The idea is that while an animal takes two years to raise, a certain synthetic leather process can be carried out in two weeks without the use of tissue engineering, the company’s initial business endeavor. Modern Meadow alters DNA base pairs to create instructions for cells to multiply and produce collagen. The produced collagen then self-assembles into a triple helix molecule, and the molecules link together to form an interwoven network of fibers. Modern Meadow uses an unspecified process to further assemble fibers into the desired material, a synthetic leather product, which can be tanned and treated for commercialization. The company has officially launched its brand Zoa, a name meant to evoke the idea of life coming into materials.

In a related field, startup Colorifix is addressing the environmental concerns associated with textile dyeing. The textile industry is the second largest polluter of water in the world, but Colorifix’s process claims to produce less than 1% of dye waste along with a 20% reduction in energy expenditure. The company uses sugar molasses feedstock to grow microorganisms in fermentation, harvests the organisms directly from fermentation, and applies them to fabrics as a part of the dyeing process. Once the dye transfer is complete, the organisms are removed from the product with high temperatures and detergents that eliminate lipids, carbohydrates, and proteins, which essentially comprise the entire organism. Colorifix promotes chemical safety but also the feasibility of its process. It is partnered with companies such as H&M and will soon be introducing its products to stores nationwide.

Figure 2. Modern Meadow animal-free leather fabrication and Colorifix textile dyeing. Sources: Modern Meadow, Colorifix, used with permission. Cell factories for biopharmaceuticals

Arguably one of the more well-known applications of biotech comes in the form of pharmaceuticals and their work in producing novel medicines. Leaders in synthetic biology are creating a new concept of “living medicines” that respond to cues from the human body itself.

Biopharma company Synlogic Therapeutics specializes in Synthetic Biotic medicines that manipulate naturally occurring microbes to achieve desired medical results. Synlogic’s work is centralized on the functions of the human microbiome, the quadrillion genes of microbial cells that inhabit the human body. The microbiome has certain established capabilities, and the pharma company means to expand these capabilities through the programming of probiotic bacteria. The chassis for Synlogic is E. coli Nissle, a proteobacteria indigenous to the microbiota. This bacteria is engineered to carry genetic circuits that allow the Synthetic Biotic medicines to detect and interpret a patient’s body signals, leading to the subsequent activation or inactivation of a specific metabolic pathway.

Synlogic currently hopes to treat patients with genetic metabolic diseases such as Urea Cycle Disorder and Phenylketonuria. However, as research continues to become available, the company intends to provide treatments for inflammatory bowel disease, Crohn’s disease, and ulcerative colitis as well.

Figure 3. Synlogic’s Synthetic Biotic medicine pipeline. Source: Synlogic Therapeutics, used with permission. Big data and artificial intelligence’s connection to biology

Artificial intelligence is an up-and-coming field, and one with many potential applications to the realm of synthetic biology. In a highly anticipated “fireside chat,” Vinod Khosla, venture capitalist, and George Church, professor of genetics at Harvard Medical School, discuss the implications of deep learning in working with big data.

Deep learning is a subfield of machine learning that uses artificial neural networks, algorithms modeled after the human brain. It is thought to be particularly useful in analyzing big data sets because it can extract high-level abstractions from large quantities of unsupervised data. Perhaps for this reason, Khosla and Church agree on the importance of integrating such deep learning tools into biotech technologies.

According to Church, “scientific literature is biased against less-than-super-exciting positive results.” However, new artificial intelligence allows for the publishing of big data sets, most of which can be negative (for example, “this RNA or protein is not expressed in this sequence”). Khosla believes that with deep learning, negative results can be just as valuable as positive results and, therefore, every piece of data contributes in some way.

While Khosla and Church delve into the many intricacies of artificial intelligence, the point of the matter is that the advancement of this technology is leading people closer and closer to re-interpreting global problems in inventive ways. Rather than making mosquitoes resistant to malaria, why not engineer human resistance? Or rather than enforcing strategies to prevent the persistence of global warming, why not instead develop methods to reverse it?

Figure 4. Left to right: Vinod Khosla and George Church. Source: SynBioBeta, used with permission. The future is synthetic biology

Amidst the enthralling presentations and tangible enthusiasm of conference participants, it’s plain to see that there is a promising future in the diversity of synthetic biology. We live in a world where scientific breakthroughs are being made daily, a world where science fiction like Gattaca is becoming a reality. Our goals are evolving to funnel a more advanced understanding of biology into new projects like DNA data storage and prosthetics made from fruits.

The amount of prospective technological change, however, begs another question: will consumers readily accept products synthesized through biological means? Emily Leproust thinks so.

“Explain cause and benefit, and people will get it,” says Leproust. “Synthetic biology has the potential to touch more parts of society and provide a greater benefit to humanity."

Continue reading SynBioBeta and the current biorevolution.

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[freesuitwhispers]

Global  Urea Cycle Disorder (UCD) Market - Growth Drivers, Opportunities And Forecast Analysis To 2019-2025

Summary - A new market study, “ Global Urea Cycle Disorder (UCD) Market - Growth Drivers, Opportunities And Forecast Analysis To 2019-2025 ” has been featured on WiseGuyReports.  

Urea cycle disorders (UCDs) are a group of orphan inherited defects of six enzymes and two transporters that constitute the urea cycle in the periportal liver cells. The age of onset symptoms vary between the different types of UCDs. UCDs often occurs during the period of infancy due to triggered postnatal catabolism. The diagnosis is made by analysis of the urine and blood for abnormal metabolites (substance produced by metabolism), and high ammonia levels. Frequent blood tests are done to monitor ammonia levels. The treatment of urea cycle disorders consists of dietary management to limit ammonia production. A liver transplant can be an effective treatment for urea cycle disorder for the proper functioning of urea cycle.

 The urea cycle disorder treatment market in the U.S. and Europe is expected to grow rapidly in the next few years. Several pharmaceutical companies are under the development process of their late stage pipeline drugs for urea cycle disorders. Factors such as, increasing healthcare expenditures in the U.S, rise in the U.S. pharmaceutical R&D expenditures and collaboration between pharmaceutical companies and research institutes have driven the growth of the U.S. urea cycle disorder treatment market. Further, upcoming launches of anti-UCD drugs would contribute to the expansion of the urea cycle disorder treatment market in the U.S. as well as in Europe.

Also Read : http://heraldkeeper.com/news/urea-cycle-disorder-ucd-market-global-share-size-trends-and-growth-analysis-forecast-to-2020-2024-513653.html

The U.S. urea cycle disorder treatment market is dominated by certain key players including Horizon Pharma Inc., Ultragenyx Pharmaceuticals Inc., Aeglea Bio Therapeutics and Synlogix. Among all the key players, Horizon Pharma ruled the U.S. UCD treatment market owing to the strong performance of its product. Horizon’ Ravicti is the market leader due its strong curative impact on the patients with UCD. The remaining key players have their products under the development process of pipeline drugs for UCDs. There would be a generic competition between the upcoming anti-UCD drugs in the coming years.

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