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Castle Creek Biosciences, Inc. is a clinical-stage cell and gene therapy company focused on developing and commercializing disease-modifying therapies for patients suffering from rare diseases for which there is a lack of available treatment options. The company`s proprietary autologous fibroblast platform potentially allows for the development of personalized, targeted and redosable cell-based gene therapy product candidates for monogenic and chronic disorders. The company`s most advanced product candidate, dabocemagene autoficel (D-Fi), is currently being evaluated in a Phase 3 clinical trial for the localized treatment of chronic wounds due to recessive dystrophic epidermolysis bullosa (RDEB). The company is also currently evaluating FCX-013 in a Phase 1/2 clinical trial for the treatment of moderate to severe localized scleroderma. In addition, Castle Creek Biosciences is pursuing discovery and potential development of early-stage novel product candidates with the goal of expanding its robust pipeline into other rare diseases and broader indications where there are significant unmet needs. The company operates an in-house, commercial-scale manufacturing facility in Exton, Pennsylvania that benefits from the validated systems and processes previously implemented at the site for manufacture of an FDA-approved cell therapy product. Castle Creek Biosciences, Inc. is a portfolio company of Paragon Biosciences, LLC.
Genyous Life Sciences is a Vancouver, BC-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
Type 1 diabetes (T1D), formerly known as juvenile diabetes, is a chronic, life-threatening disease that affects millions of people worldwide. In the United States, 30,000 new cases are estimated every year with half of those cases diagnosed in young children. Type 1 diabetes is an autoimmune disease in which the patient`s immune system goes awry and attacks and destroys the pancreatic beta cells. Beta cells are responsible for regulating blood sugar (glucose) levels by producing precise amounts of the essential hormone insulin. The discovery of injectable insulin in the 1920s changed T1D from a uniformly fatal disease with a life expectancy of months to one that could be carefully managed for decades through multiple daily blood glucose measurements and insulin injections. However, insulin injections are not a cure and patients face a lifetime of difficult disease management and serious complications including kidney failure, blindness and nerve damage. Despite nearly a century passing since the discovery of insulin, insulin injection remains the only treatment available to patients. Semma Therapeutics was founded to develop transformative therapies for patients who currently depend on insulin injections. Recent work in the laboratory of Professor Douglas Melton led to the discovery of a method to generate billions of functional, insulin-producing beta cells in the laboratory. These cells develop in islet-like clusters grown from stem cells. Initial preclinical work in animal models of diabetes has shown that transplantation of these cells are sufficient to control blood glucose levels. This breakthrough technology has been exclusively licensed to Semma Therapeutics for the development of a cell-based therapy for diabetes. Ongoing research at Semma Therapeutics is focused on combining these proprietary cells with a state-of-the-art cell delivery and immune protection strategy that can protect these cells from the patient`s immune system and allow the beta cells to function as they do in non-diabetic individuals. Implantation of the beta cell-filled device has the potential to provide a true replacement for the missing beta cells in a diabetic patient and would not require patient immunosuppression. Semma Therapeutics is working to bring this new therapeutic option to the clinic and improve the lives of patients with diabetes.
Scholar Rock is a clinical-stage biopharmaceutical company focused on the discovery and development of innovative medicines for the treatment of serious diseases in which signaling by protein growth factors plays a fundamental role. Our most advanced product candidate, apitegromab (SRK-015), a selective first-in-class inhibitor of the activation of the growth factor myostatin in skeletal muscle, is currently in development for the treatment of spinal muscular atrophy, or SMA. We are currently evaluating apitegromab in our TOPAZ Phase 2 proof-of-concept trial in patients with later-onset (Type 2 and Type 3) SMA. Positive six-month interim analysis results were announced in October 2020 and top-line data for the full 12-month treatment period are anticipated in the second quarter of 2021. In addition, we are conducting our DRAGON Phase 1 proof-of-concept trial for SRK-181, a highly specific inhibitor of latent TGFβ1 activation, in patients with locally advanced and metastatic solid tumors. We believe SRK-181 has the potential to overcome resistance and meaningfully increase the number of patients who may benefit from checkpoint inhibitors, such as anti-PD-1 or anti-PD-L1 therapies. Utilizing our proprietary platform, we are creating a pipeline of novel product candidates with the potential to transform the lives of patients suffering from a wide range of serious diseases, including neuromuscular disorders, cancer, fibrosis and anemia.
Pionyr Immuntherapeutics (formerly Precision Immune Inc.) is developing cancer immunotherapies that target the tumor microenvironment to enhance the body`s antitumor immunity. The company is exploiting novel target discovery and antibody generation platform technologies to create the next generation of immuno-oncology therapeutics. The company`s approach, Myeloid Tuning™, is designed to enhance the immune system`s anti-tumor response by altering the cellular infiltrate of the tumor microenvironment with high specificity.