Ultragenyx Pharmaceutical develops therapeutics for rare diseases, sometimes referred to as Orphan products. Ultragenyx is a efficient and effective development strategies are essential for success in the rare disease space, in which information may be incomplete or ambiguous, and few development precedents exist. The management team's experience in selecting and developing products with clear mechanisms of action for untreated rare diseases will help Ultragenyx create life-changing therapeutics by leveraging existing yet undeveloped science for these little-known indications.
Amlogenyx is a novel gene therapeutics company that focuses on developing lysosome-based treatments for Alzheimer's disease. It is a spin-off from Ultragenyx Pharmaceutical.
GeneTx Biotherapeutics is a biotechnology company focusing on developing therapeutics for the treatment of Angelman syndrome. It also offers an antisense therapy for treating the rare genetic disorder that causes developmental delay, impaired motor function, loss of speech, and epilepsy. Founded in 2017, GeneTx Biotherapeutics is headquartered in Downers Grove, Illinois.
Solid Biosciences Inc. is a life science company focused on developing therapies for Duchenne muscular dystrophy (DMD), a severe genetic muscle-wasting disease primarily affecting boys. The company's lead product candidate, SGT-001, is a gene transfer therapy currently undergoing a Phase I/II clinical trial aimed at restoring functional dystrophin protein expression in patients’ muscles. DMD is a progressive and ultimately fatal condition, with an estimated prevalence of 10,000 to 15,000 cases in the United States. In addition to SGT-001, Solid Biosciences is advancing the Anti-LTBP4 program, which targets the LTBP4 protein to reduce fibrosis and inflammation. The company also develops biomarkers, sensors, and the Solid Suit program, which includes wearable assistive devices designed to provide functional and therapeutic benefits. Established in 2013 and headquartered in Cambridge, Massachusetts, Solid Biosciences collaborates with Ultragenyx to enhance its gene therapy initiatives for DMD.
Dimension Therapeutics is a gene therapy company focused on developing novel treatments for rare diseases. Their team comprises biotech industry veterans and thought leaders in gene therapy and rare diseases. The company is focused on advancing its platform of gene therapy programs in rare diseases through clinical development, starting with lead programs in hemophilia, and building out a world-class product engine for adeno-associated virus (AAV) therapeutics. In conjunction with its launch, Dimension has entered into an exclusive license and collaboration with REGENX Biosciences. REGENX holds exclusive rights to a portfolio of over 100 patents and patent applications pertaining to its NAV vector technology and related applications. Through its license and collaboration with REGENX, Dimension has acquired preferred access to NAV vector technology and rights within REGENX product programs in multiple rare disease indications. Dimension has completed a Series A financing led by Fidelity Biosciences.
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