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Find Drugs for Rare Diseases and Disorders in Preclinical Development
GC1126A is a novel ADAMTS13 mutein designed to evade autoantibodies with an extended half-life, which is investigated for the treatment of thrombotic thrombocytopenic purpura.
Under the agreement, Alexion expand its rare disease gene therapy portfolio by gaining Pfizer’s early-stage preclinical gene therapy programmes. The agreement furthers Alexion's commitment to advancing next-generation genomic medicines.
Cannabidiol, a chemical compound from the cannabis plant, that help improvement in social and motor problems, a reduction in epilepsy and an increase in life expectancy in Leigh syndrome mouse models.
The funding will be used for a novel Adeno-Associated Virus (AAV) gene therapy, GA-002, aimed at curing GNE Myopathy (GNEM) or Hereditary Inclusion Body Myopathy (HIBM).
Under the agreement, Alexion will expand its rare disease gene therapy portfolio by gaining Pfizer’s early-stage preclinical gene therapy programmes. The agreement furthers Alexion's commitment to advancing next-generation genomic medicines.
Under the terms of the agreement, Sangamo has received a non-exclusive license to combine a Voyager TRACER capsid with Sangamo’s ZF-TRs (zinc finger transcriptional regulator) designed to treat prion disease.
Two new gene therapy trials at UT Southwestern and Children’s Health funded through the consortium will test treatments for multiple sulfatase deficiency and Charcot-Marie-Tooth disease type 4J (CMT4J). Each clinical trial will include between eight and 12 patients.
The agreement aims for the clinical development of NephroDI’s asset, NDI-5001, a novel kidney-targeted small molecule AMPK activator, as a potential therapy for X-linked congenital Nephrogenic Diabetes Insipidus (NDI).
The financing will fund completion of a first-in-man proof of concept study in subjects with Usher1B and further development of AAV intein program and entry into the clinic for Stargardt disease with AAVantgarde's two proprietary, AAV-based large gene delivery platforms.
Company will donate licenses to NAV® AAV8 and NAV® AAV9 from its NAV® Technology Platform to enable AMP® BGTC's development of these programs aimed at addressing ultra rare diseases i.e. Mucopolysaccharidosis type IVA (MPS IVA), also known as Morquio syndrome.
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