gene therapy

In Tuesday’s pre-market trading, ANEW Medical, Inc. (NASDAQ:WENA) saw its shares surge nearly 80% following an announcement about the advancement of its patented Klotho gene therapy program for neurodegenerative disorders. What Happened: ANEW, a biopharmaceutical company that specializes in...

An 18-month-old British child has regained her hearing through a pioneering gene therapy, marking a significant stride in the treatment of deafness. What Happened: Opal Sandy, who was born with a rare genetic condition that left her completely deaf, has now been able to hear without the need for...

A young patient died due to cardiac arrest after receiving Pfizer Inc’s (NYSE:PFE) experimental gene therapy in a mid-stage trial for Duchenne muscular dystrophy (DMD), the company told in a letter to Parent Project Muscular Dystrophy. The boy was enrolled in Phase 2...

Verve Therapeutics Inc (NASDAQ:VERV) announced an update from the Heart-1 Phase 1b trial of VERVE-101. VERVE-101 is being evaluated in patients with a genetic condition that leads to high cholesterol levels, which raises the risk of heart disease. The treatment is supposed to permanently...

Astellas Pharma Inc (OTC: ALPMF) (OTC: ALPMY) decided to license and advance a gene therapy from Kate Therapeutics for a debilitating muscle disorder known as X-linked myotubular myopathy (XLMTM). Preclinical gene therapy candidate KT430 will seek to address XLMTM, a rare and...

On Friday, FDA's Cellular, Tissue and Gene Therapies Advisory Committee (CTGTAC) voted 8 to 6 in support of accelerated approval of Sarepta Therapeutics Inc's (NASDAQ: SRPT) SRP-9001 (delandistrogene moxeparvovec) for ambulatory patients with Duchenne muscular dystrophy. The...

Prices for incoming sickle cell disease gene therapies from Vertex Pharmaceuticals Inc (NASDAQ: VRTX)/CRISPR Therapeutics AG (NASDAQ: CRSP), dubbed exagamglogene autotemcel (exa-cel), and Bluebird bio Inc’s (NASDAQ: BLUE) lovotibeglogene autotemcel (lovo-cel) at $2...

Rocket Pharma (NASDAQ: RCKT) CEO Gaurav Shah is on a mission to "change the narrative" when it comes to rare diseases. "We are enthusiastic about the potential of [our] strategy to hopefully bring much-needed therapies to patients that wouldn’t otherwise have them," he...

Major Wall Street indices closed mixed on Wednesday after troubles at Swiss lender Credit Suisse Group AG (NYSE:CS) brought back fears of a potential contagion and dampened hopes of a smaller rate hike by the Federal Reserve later this month. The Nasdaq Composite closed 0.05% higher while the S...

Regenxbio Inc (NASDAQ: RGNX) announced additional interim data from the ongoing Phase 2 ALTITUDE trial of RGX-314 for diabetic retinopathy (DR) without center-involved diabetic macular edema (CI-DME). At six months, patients treated with RGX-314 demonstrated clinically meaningful...

Ocugen Inc (NASDAQ: OCGN) shares traded marginally higher by 1.22% at $1.66 during Wednesday's after-hours session. The company announced during after-hours trading that the Independent Data and Safety Monitoring Board (DSMB) for the OCU400 Phase 1/2 clinical trial completed a review...

Mizuho initiated coverage on Ocugen Inc (NASDAQ: OCGN) with a Buy rating and a price target of $5, reflecting an upside of almost 115%. Mizuho views Ocugen as a high-risk, high-reward opportunity. While the lead asset, a COVID-19 vaccine (Covaxin), offers potential near-term...

The FDA has approved Bluebird bio Inc's (NASDAQ: BLUE) Zynteglo (betibeglogene autotemcel), also known as beti-cel, a one-time gene therapy custom-designed for beta‑thalassemia. The approval covers adult and pediatric patients who require regular red blood cell (RBC)...

Homology Medicines Inc (NASDAQ: FIXX) announced the details of HMI-204, its optimized, in vivo, one-time gene therapy candidate for metachromatic leukodystrophy (MLD).  Following a single I.V. administration in the MLD animal model, the candidate crossed the blood-brain barrier...

Small biotech Freeline Therapeutics Holdings plc (NASDAQ: FRLN) hopes new long-term data will help boost its case. The company announced that the New England Journal of Medicine (NEJM) published positive data, expanding upon previously announced long-term follow-up data for up to 3....