Genetic Disorder

Quoin Pharmaceuticals Ltd (NASDAQ: QNRX) has entered into an exclusive License and Distribution Agreement with Genpharm Services for QRX003 for Netherton Syndrome. Under the agreement terms, Genpharm has exclusive rights to commercialize QRX003 in the Middle East and North...

Quoin Pharmaceuticals Ltd (NASDAQ: QNRX) has entered into an exclusive License and Distribution Agreement with AFT Pharmaceuticals Ltd for QRX003 for Netherton Syndrome. Under the terms of the agreement, AFT has exclusive rights to commercialize QRX003 in Australia and New...

Mammoth Biosciences unveiled a nearly $700 million deal with Vertex Pharmaceuticals Incorporated (NASDAQ: VRTX) to develop in vivo gene therapies for two genetic diseases. Vertex is putting down $41 million upfront, including a convertible note, to use Mammoth's CRISPR systems. And...

Stealth BioTherapeutics Corp (NASDAQ: MITO) has received a Refusal to File letter from the FDA regarding its marketing application for elamipretide, a mitochondria-targeted therapy for Barth syndrome. Barth syndrome is an ultra-rare genetic condition characterized by cardiac...

PTC Therapeutics Inc (NASDAQ: PTCT) presented a new analysis of five-year results of gene therapy, PTC-AADC. The data exhibited improvements in children with aromatic L-Amino acid decarboxylase (AADC) deficiency. AADC deficiency is a rare genetic disorder that causes severe...

Eli Lilly And Co (NYSE: LLY) is doubling down on RNA research as it pens another pact with a quiet RNA editing player focused on neuroscience, ProQR Therapeutics N.V. (NASDAQ: PRQR). In May, Lilly teamed up with MiNA Therapeutics to tap its small activating RNA (...

AstraZeneca plc (NASDAQ: AZN) reported high-level results from the FoCus Phase 3 trial evaluating ALXN1840 in Wilson disease. The study met the primary goal of improving the daily mean copper mobilized from tissues over 48 weeks. Wilson disease is a genetic condition in which the...

Stealth BioTherapeutics Corp (NASDAQ: MITO) has submitted an elamipretide marketing application to the FDA for Barth syndrome. The submission is based on results from the SPIBA-001 Phase 3 Retrospective Natural History Control Trial, which compared data from the TAZPOWER Phase 2/3...

The Brazilian Health Regulatory Agency has approved PTC Therapeutics Inc's (NASDAQ: PTCT) Waylivra (volanesorsen) as the first treatment for familial chylomicronemia syndrome (FCS).  The approval is based on the Phase 3 APPROACH study results and the APPROACH Open-Label...

Cyclo Therapeutics Inc (NASDAQ: CYTH) has announced new data from its Phase 1 extension study evaluating Trappsol Cyclo Niemann-Pick Disease Type C1 (NPC), a genetic disorder characterized by the abnormal accumulation of cholesterol in cells.  Efficacy as measured NPC Severity...

European Union has approved AstraZeneca plc's (NASDAQ: AZN) Koselugo to treat children with neurofibromatosis type 1 and plexiform neurofibromas. Neurofibromatosis type 1 is characterized by soft lumps on and under the skin and skin pigmentation. The approval was granted by the...