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Krystal Biotech’s Vyjuvek has been awarded US Food and Drug Administration (FDA) approval to make it the first topical gene therapy for the treatment of wounds in patients with the rare, often debilitating skin disease dystrophic epidermolysis bullosa (DEB). As a topical treatment, it is also the first readily redosable gene therapy.
Discovery could lead to new screening tests and potential treatments Credit: Cincinnati Children’s Two common variants in the KIF3A gene increase the risk of young children having a dysfunctional skin barrier and developing the skin condition atopic dermatitis.
For instance, Vyjuvek , the first FDA-approved gene therapy for DEB, is priced at $24,250 per vial. a biotech company specializing in the development and commercialization of genetic medicines for rare diseases, announced FDA approval for Vyjuvek for the treatment of DEB.
A test that monitors blood levels of DNA fragments released by dying tumor cells may serve as an accurate early indicator of treatment success in people in late stages of one of the most aggressive forms of skin cancer, a new study finds. Led by NYU Grossman School of Medicine and Perlmutter Cancer Center researchers, […].
Metabolic disorders are top therapy areas Analysis curated by GlobalData [5] shows the top three therapy areas for DCT adoption in the first quarter of 2022 were metabolic disorders, followed by central nervous system (CNS) and genetic disorders. However, there is still some use of remote patient monitoring technologies.
GPP occurs in individuals with specific gene variations as it is a genetic disease , and variations in the genes IL36RN and AP1S3 are known to cause it. The role of the IL-36 receptor was confirmed in loss-of-function mutation studies in the IL-36 receptor antagonist gene. Waldman Department of Dermatology.
The precise cause of psoriasis is unknown, but risk factors include genetics and a family history of the condition, heavy alcohol consumption, stress, smoking and dry or cold weather. At Dermavant, we are committed to advancing novel, patient-focused innovation in immuno-dermatology. Clinical Trials of Vtama.
A new drug designed to work on cancers with an altered BRAF gene has shown promise in an early patient trial presented at the 32nd EORTC-NCI-AACR [1] Symposium on Molecular Targets and Cancer Therapeutics, which is taking place online. The BRAF gene is involved in telling healthy cells when to grow and form new cells, […].
TSX: CTX) (OTC US: CRRTF) (“Crescita” or the “Company”), a growth-oriented, innovation-driven Canadian commercial dermatology company with in-house research & development (“R&D”) and manufacturing capabilities, today announced that it has entered into an exclusive agreement with Juyou-Biotechnology Co.
Most recently, Cadavid served as senior vice president and Head of Clinical Development at Fulcrum Therapeutics where he led the development of multiple small molecules for the treatment of genetically defined rare diseases. Prior to SpringWorks, Smith was the executive vice president of Gene Therapy at Bamboo Therapeutics.
The companies include a diverse range engaged in innovative approaches in areas like oncology, genetic medicines, inflammatory diseases, dermatology and cardiorenal therapy. Check out this article to learn about the biotech companies that went public this year.
Pfizer’s Rare Disease late-stage pipeline currently includes three gene therapy programs that, if successful, are expected to gain regulatory approval by the end of 2023, with an additional pipeline of 10 preclinical initiatives that are at various stages of maturity. Inflammation and Immunology.
Garrido, CNIO The sophisticated human immune system has evolved to become an effective protection system against a great number of diseases, cancer being one of them. The immune system recognises and destroys cancer cells using a monitoring process called immunological surveillance.
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