gene therapy

Investors are closely watching stock trades by members of Congress, where committee roles and trade timing can raise red flags—or attract copycats. That scrutiny has intensified after one lawmaker disclosed purchases in a microcap company valued at less than $5 million, an unusually small and risky...

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...