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Rare Skin Diseases: Key Companies Making A Difference

Dermatology indications are a promising area of focus for future gene therapy in dermatology. The following list gives a brief detail of some of these pharmaceutical companies, along with their lead products.

Abeona Therapeutics

Abeona Therapeutics is the leader of the rapidly advancing field of genetic medicine. It is a fully integrated gene and cell therapy company. Its competence across discovering novel cell and gene therapies, manufacture, and R&D has led to the production of new drugs for patients in need.

Patients suffering from RDEB, a rare connective tissue disorder that has no valid treatment, face severe epidermal wounds that impact their longevity. Abeone’s lead asset EB-101 is a  gene-corrected, autologous cell therapy that addresses RDEB.

Castle Creek Biosciences

Rare skin diseases lack available treatment options for suffering patients. Castle Creek Biosciences, Inc. - a clinical-stage cell and gene therapy company, works on developing and commercializing disease-modifying treatment options for this segment. The company uses technology that is compatible with the unique biology of each patient. It does so by extracting fibroblast cells from a patient’s skin and then creating localized treatments.

The company has two lead therapies undergoing clinical development. 

Therapy 1 includes FCX-013 that is to be used as an autologous cell-based, disease-modifying gene therapy.  It utilizes lentiviral vectors to deliver functional matrix metalloproteinase 1 genes (MMP-1) to patients with moderate to severe localized scleroderma.

Therapy 2 includes D-Fi (dabocemagene autoficel, formerly known as FCX-007) developed as an autologous cell-based, disease-modifying gene therapy. It addresses functional type VII collagen protein (COL7) deficiency in patients that have autosomally recessive or dominant dystrophic epidermolysis bullosa (DEB).

Krystal Biotech

Krystal Biotech, Inc. is a company that is in the clinical stage, working to develop effective and innovative treatments for skin diseases. It has been working on expanding the use of its proprietary Skin TARgeted Delivery (STAR-D) platform to target and treat many skin conditions. It has also been developing topical and intradermal “off-the-shelf” novel therapies for treating rare dermatological indications.

Beremagene geperpavec (“B-VEC,” previously “KB103”) is Krystal’s lead therapy prospect, currently in Phase III stage of development, that uses gene therapy for treating all forms of dystrophic epidermolysis bullosa. 

Conclusion

Apart from the above-mentioned companies, there are many more Key Companies In Gene Therapy For Dermatology working continuously to satisfy the unmet demands of treatment options for rare conditions in skin diseases. The future of gene therapy in dermatology looks promising since many biotech and pharmaceutical companies are developing novel gene therapies products for various skin diseases. Thus, we may see many more advancements in this subject shortly.

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Bone Metastasis: A Cancer Caused By Cancer

Medical Diagnosis has advanced monumentally in recent years. Regardless, prior detection of Bone Metastasis can be complicated, and the detection of its presence often means that the patient has reached an advanced disease stage and has only a little time left with a few likely treatment options.

The foremost goal of these treatment options is not to overcome the condition but rather to relieve the associated pain, prevent further bone complications, and sustain a good quality of life. Upon diagnosis of the first metastatic bone cancer, the life expectancy is often around 6-48 months.

What Is Bone Metastasis?

Bone Metastasis is most commonly observed in patients suffering from lung, prostate, and breast cancer. It occurs in advanced cancer patients when cancerous cells from the primary tumor move to the bone.

Its symptoms include weakness in legs or arms, broken bones, bone pain, bowel and urinary incontinence, and hypercalcemia (a condition where high levels of calcium are seen in the blood), which lead to nausea, constipation, vomiting, and confusion.

Since this condition has adverse clinical consequences, a prompt and precise diagnosis is thus necessary. The diagnosis usually includes looking for symptoms, signs, and imaging. Laboratory tests include Plain X-ray, Computed Tomography (CT), and Radionuclide Bone Scintigraphy that can be used as the imaging methods for detecting Bone Metastasis.

What Are The Treatment For Bone Metastasis?

According to the reports, breast cancer has the highest prevalent cases of Bone Metastasis, with around 121,603 reported cases in 2020, within the metastatic group in the United States, followed by lung cancer, prostate cancer, and other solid tumors.

Further analysis shows that the number of cases of Bone Metastasis established on tumor type could climb by 2030 due to the increasing incidence of cancer globally, which could make a positive shift in the Bone Metastasis market.

A study of the Bone Metastasis Treatment Market describes the current therapies, the key companies, and the emerging pipeline in further detail. The study also indicates the progression of developmental stages and the survival rate of Bone metastasis as observed in many patients over the years.

Bone Metastasis is hardly ever cured, and the only way of treatment is through disease control, performed using systemic anticancer therapies built on multidisciplinary supportive care.

This care includes using denosumab or bisphosphonates (a bone-targeting agent) to prevent skeletal morbidity and inhibit tumor-related osteolysis. Appropriate local treatments such as specialist palliative care, radiation therapy, orthopedic surgery are further used to reduce the impact of metastatic bone disease on bodily functioning.

The Future

It is necessary to develop novel strategies that improve the patient's quality of life by averting skeletal disease and palliating established skeletal events. In addition, more studies are vital for understanding the interaction between the tumor cell and bone microenvironment so that effective new therapeutic interventions can be developed.

The entry of important pharmaceutical companies pioneering novel effective therapies, the discovery of new biomarkers for diagnosis, and R&D, shall propel the Bone Metastasis market landscape and treatment frame. With growing awareness of Bone Metastasis among people, the complete Bone Metastasis treatment landscape could change significantly in the coming years.

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Understanding Learning Disability And Its Types

Learning disability is a disorder that lasts throughout a person’s life and has no cure. It prohibits a person from acquiring a new skill and comprehending complex information, further hampering their ability to be self-reliant and use their skills productively.

If recognized early, interventions can be more effective, and the person affected need not face extended problems or go through low self-esteem later in life. For early identification, however, it is necessary to have a general awareness of the disability and its types.

What Learning Disability Is.

A Learning Disability (LD) is a neurological condition resulting from neurobiological factors and genetics, affecting the brain’s capability to process information and execute functions. It is to be considered that a learning disability is not an intellectual disability, vision problem, hearing loss, emotional or behavioral disturbance but instead more of learning and acquiring a skill and using it effectively.

The learning disabilities may be in mathematics, reading, or oral or written language, and it is commonly identified at an early age however, in some cases, it may not be identified until adulthood or at later life. According to the National Adult Literacy and Learning Disabilities Center, “ nearly 60% of adults with severe literacy problems have undetected or untreated learning disabilities”. The severity and characteristics of LD can vary from person to person.

Causes Of Learning Disability

A learning disability can be caused by many factors. It can be due to some prenatal issues or difficulties faced during birth or due to genetics. Apart from that, diseases or other similar conditions such as Autism Spectrum Disorder, Epilepsy, Meningitis, age, or traumatic brain injuries are the chief causes for developing a learning disability.

Treatments For Learning Disability

Presently, there is no definite medication or medical treatment available to treat learning disabilities. However, the Learning Disability Treatment Landscape is evolving with the emergence of Digital Assistant Technologies.

Level Of Severities In Learning Disability

In severe cases, the person experiences difficulties in many academic fields, and the person needs rigorous specialized teaching to overcome these.

In average cases, the person might experience difficulties in learning more than two academic subjects, while in mild cases, the person may have hardships in learning one or two academic areas only.

Learning Disabilities Types

Dysgraphia - Dysgraphia is more prominent in males than in females. In this type, the affected person finds it challenging to put their thoughts on paper or drawing. Most people with dysgraphia are unaware that they are affected by it.

Dyslexia - This is the most common type of learning disability. It is related to acquiring and processing language. The affected person may face challenges in reading, accurate and fluent word recognition, writing, and understanding literature.

Dyscalculia - Dyscalculia is a disability associated with mathematical calculations. The affected person finds difficulties in learning numbers, using concepts, using mathematical symbols, using mathematical functions and facts, and reasoning.

Nonverbal learning disabilities - These disabilities are classified into three segments - social abilities, visual-spatial organizational memory, and motor skills. Here, the affected person finds difficulties in decoding nonverbal behaviors or social cues. The person may also struggle with understanding facial expression and voice tone, body language, and have poor coordination skills.

Auditory processing disorder - APD is also referred to as Central Auditory Processing Disorder (CAPD). It is closely linked to dyslexia as approximately 50% of children with dyslexia also have APD. The affected person faces difficulties in recognizing the correct order of sounds, processing sounds, or is unable to filter different sounds.

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Can Gene Therapies Revolutionize The Treatments In Dermatology?

Improvements in healthcare have been changing lives regularly, and one of the most remarkable changes has been brought by gene therapy. Usually, in Gene Therapy, an individual’s gene is modified to cure a disease that is often rare or a type of autoimmune disorder. A genetic impairment does not always lead to an autoimmune disorder; sometimes, it further impacts the epidermis and the dermis. Earlier, rare skin diseases were considered impossible to heal, but the new developments in genetic therapeutics have given rise to new possibilities. Since the skin is superficial, easily manipulatable, and a versatile organ, it is one of the easiest targets for gene therapy.

Gene therapy is considered revolutionary for the treatment of many hereditary dermatological disorders like Epidermolysis bullosa and its 3 subtypes - Dystrophic epidermolysis bullosa (DEB), Epidermolysis bullosa simplex (EBS), and Junctional epidermolysis bullosa (JEB). Other rare dermatological conditions include Netherton Syndrome, Pachyonychia congenita, Ichthyosis, Xeroderma pigmentosum, Porphyra, Sjogren-Larson syndrome, etc. These uncommon genetic skin disorders cause many blisters, severe lesions, and rashes on the skin. With Gene correction techniques, these impaired genes are targeted to modify the already existing gene expression and transfect them with the healthy alternative.

Genetic Engineering, along with treating rare skin diseases, can also treat dermatological conditions that do not involve genetic mutation at all, such as burns and wounds. Gene therapy offers valuable remedy options for treating conditions like diabetic ulcers, refractory burn wounds, vascular ulcers.

Skin Cancer is one of the most widespread cancers, and 1 in every 5 Americans experience it after 70 years of age. Other studies show that in the USA, 1-2 people die of Skin cancer every hour. However, due to the evolution in gene therapy, skin cancer can now be treated to some extent by the application of recombinant DNA technology.

Many key pharma and biotech companies are working incessantly to develop gene therapies associated with dermatology diseases, including Castle Creek Biosciences, Krystal Biotech, Almirall, Abeona Therapeutics, Tyris Therapeutics, Fibrocell Science, Sterna Biologicals, Amryt Pharma, Temprian Therapeutics, Holostem Terapie, Avita Medical, Azitra, Novartis, and several others.

The development of Gene Therapies in Dermatology is an exciting and promising subject to look towards.

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The Finest Applications Of Robots In The Healthcare Industry.

Humans are not ideal for repetitive and monotonous work, and therefore they are being replaced by robots. However, with the advent of Artificial intelligence (AI), the once limited scope of Robot employment is widening. Robots lower production costs and increase productivity simultaneously. As per the International Federation of Robotics, 74 robot units per 10,000 employees is the new average of global robot density in the manufacturing industry alone.

Now, other sectors, such as laboratory research, earth and space exploration, transport, and many more, are highly utilizing robots. The Healthcare Industry has undertaken the adoption of robots for accomplishing different (and often complex) tasks. Clinical training, complicated surgeries, personal care, medicine dispensing, etc. are just a few among the many applications in which AI and Robotics have addressed and provided successful solutions.

According to the International Federation of Robotics, sales of medical robots have risen by 73% compared to 2016 to 2,931 units in 2017, deeming for a share of 2.7% of the total unit sales of professional service robots. Robot-assisted surgery or therapy is the chief application of Robots in the Healthcare industry. However, this requirement for robots has been slowly increasing for other applications as well.

The following list discusses the peak applications of robots in the Healthcare Industry.

Providing Patient Care Services.

Care Robots are in high demand, usually in the aging population and patients with dementia and other mental diseases. Care robots fetch food and water, give mobility and transportation support, set medication reminders, and perform routine body check-ups (like temperature, blood pressure, sugar levels).

Sanitation And Disinfection.

Lack of hygiene and poor sanitation leads to many diseases, for which it is necessary to perform regular disinfection and sanitation processes. Hospitals deploy specialized robots to scan the hospital and execute the air circulating and surface disinfection processes. UV sterilization robots are one such example that eliminates bacteria, viruses, and other harmful microorganisms that can cause infections.

Distribution Of Medicines And Hazardous materials.

Dispensing medicine rapidly and accurately can be crucial during emergencies, especially in large hospital environments that can expect to see an influx of patients at any given moment. This dispensing of medicines can also involve handling sensitive liquids or viscid materials. To carry out these tasks in an error-free way, hospitals use Dispensing robots.

Training For Clinical Situations.

Clinical Training Robots produce a realistic simulation in which healthcare providers can enhance their skills and knowledge. Manufactured by Gaumard Scientific, Pediatric Hal is an Artificial Intelligence that can mimic a five-year-old child and displays symptoms of cardiac arrest and arrhythmia. It also has human-like responses such as facial expressions, mimicking crying, imitating rapid breathing, thus providing a real “working under pressure” condition to the healthcare trainees.

Surgical Applications.

Surgery is one of the finest applications of robots in the Healthcare industry so far. A surgical robot can provide a 3-D view along with high-definition and magnification capabilities. Also, Robot-assisted surgeries are “minimally invasive” than traditional surgical procedures and are more precise and flexible. Surgical Robots also reduce hospital stay and cause fewer complications. They are used extensively for surgical procedures such as bariatric, orthopedic, urological, and gynecological.

The utilization of Robots in Healthcare is a broad subject to cover. There are many benefits and drawbacks of employing robots in this particular industry. Because they are less invasive, Robots reduce blood loss and improve the recovery time for the patients. On the other hand, the chances of errors and failures associated with robots are always a possibility. However, with each new application and feature, healthcare robots are expected to improve in accuracy, quality, operational efficiencies, and safety.

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Heart Failure And The Role Of Healthcare Companies In Treating The Global Health Problem

Heart failure is a universal health problem. With a worldwide prevalence of approximately 26 million, it has become a global pandemic causing considerable healthcare expenditure, a dismal quality of life, and morbidity.

The symptoms of heart failure can be abstruse at first, frequently misdiagnosed as typical signs of aging. However, with each progressing stage, these signs become more clear. So what is Heart failure? What are its causes and symptoms? And most importantly, what are the current diagnostics and treatment methods utilized for it?

What is Heart Failure?

Heart failure (HF) is an illness in which the heart muscle cannot pump blood properly, causing blood and fluid to accumulate in the lungs, resulting in shortness of breath. Its prime causes are certain conditions like narrowed arteries in the heart (coronary artery disease) or high blood pressure, which gradually weaken or stiffen the heart, making it unable to fill and pump blood properly.

The most common symptom of HF is vessel blockage caused by extra fluid or congestion in the lungs, which is then carried further to other parts of the body. It happens in three stages - Congestion of the lung and left side of the heart, congestion of the right heart, and low cardiac output.

The most common causes of heart failure include lowering of the left ventricular myocardial utility and the dysfunction of the endocardium, pericardium, myocardium, heart valves, or large vessels, sometimes individually and sometimes in combination. Other pathogenic mechanisms leading to heart failure include - irregular myocyte calcium cycling, excessive neurohumoral stimulation, extreme or inadequate extracellular matrix production, augmented hemodynamic overload, ischemic dysfunction, enhanced apoptosis, ventricular remodeling, and genetic mutations.

Diagnosis of Heart Failure.

In a condition like HF, the heart does not stop working but instead requires some assistance to function correctly. Even with a confirmed diagnosis, a person can still lead a healthy life with proper care and management. Various parameters are considered for its diagnosis.

Blood tests diagnosis include parameters such as analyzing complete blood count, urinalysis, blood urea nitrogen, a complete metabolic profile for serum electrolytes, serum creatinine, fasting lipid profile, glucose, liver function tests, and thyroid-stimulating hormone levels.

Other heart failure diagnostic tests include a chest X-ray, Computerised tomography (CT) scans, transthoracic echocardiography (TTE), and magnetic resonance imaging (MRI), Echocardiogram, Myocardial biopsy, Stress test, Coronary angiogram, Ejection fraction, etc.

Current Treatments For HF.

Heart failure is a worldwide morbidity, increasing as the world’s population ages, and without treatment, patients with HF have a very poor prognosis. The primary goals of heart failure treatment are to reduce mortality, improve prognosis, and relieve symptoms and reduce morbidity by reversing or slowing cardiac and peripheral dysfunction.

Many pharma and MedTech companies work in the space to deliver the best class therapeutics and medical devices to lower down the burden of heart failure.

Pharma companies like Merck, AstraZeneca, Amgen, and Novartis, produce US FDA-approved drugs like Verquvo, Farxiga (dapagliflozin), Corlanor (ivabradine), and Entresto (sacubitril/valsartan), respectively.

MedTech companies such as Boehringer Ingelheim, Edwards Lifesciences, Eli Lilly, Merck, Bayer, Abbott, Medtronic, B. Braun Melsungen AG, Oscor, Abiomed, Teleflex Incorporated, etc. provide their products for the heart failure market. Among these, Boston Scientific Corporation, which is one of the renowned MedTech companies in the world, offers its four major products, namely, S-ICD™ System, VALITUDE™ X4 CRT-P, Maverick2 ™ PTCA Balloon Catheters, and ACCOLADE™ Pacing Systems.

For more thorough information on the current and upcoming treatments for HF, read this comprehensive article made by Delveinsight on the Heart failure market.

Conclusion.

Heart failure is a complex disease that has long been causing morbidity and mortality in developing as well as developed countries. In the early stages of HF, standardized medical therapy has been effective. However, current medical treatment for heart failure only alleviates symptoms, delays deterioration, and modestly prolongs life. But with advancements in science, an effective cure for HF seems feasible.

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Can Digital Therapeutics Be Effective In Treating Depression?

Current treatments for Depression come with various limitations. Using Digital Therapeutics as a new option for treating Depression has proven effective in addressing these limitations. This microblog talks about what it is and how it helps patients cope with Depression.