gene therapy

Bluebird Bio Inc (NASDAQ: BLUE) completed the rolling submission of its marketing application to the FDA seeking approval for betibeglogene autotemcel (beti-cel) gene therapy. The application covers adult, adolescent, and pediatric patients with β-thalassemia who require...

Novartis AG (NYSE: NVS) has acquired Arctos Medical, adding a preclinical optogenetics-based AAV gene therapy program and Arctos’ proprietary technology to its ophthalmology portfolio.  Deal terms were not disclosed. Related: Why Did Deutsche Bank Downgrade This Big...

The FDA has signed off Intellia Therapeutics Inc's (NASDAQ: NTLA) investigational new drug (IND) application for NTLA-5001. Related Content: Intellia Shares Shoot Higher On Promising Biomarker Data From CRISPR/Cas9 Genome Editing Candidate. NTLA-5001 is an autologous T cell...

Astellas Pharma Inc (OTC: ALPMF) has seen the fourth death in its experimental gene therapy trial gained through a $3 billion buyout of Audentes Therapeutics Inc. The Aspiro trial was testing AT132, gene therapy for a rare neuromuscular disease, X-linked Myotubular Myopathy (...

AbbVie Inc (NYSE: ABBV) and Regenxbio Inc (NASDAQ: RGNX) have partnered to develop and commercialize RGX-314, potential one-time gene therapy for wet age-related macular degeneration (wet AMD), diabetic retinopathy (DR), and other chronic retinal diseases.  RGX-314 is...

The FDA has granted both Orphan Drug Designation and Rare Pediatric Disease Designation to Decibel Therapeutics Inc (NASDAQ: DBTX) lead gene therapy product candidate, DB-OTO, for otoferlin-related congenital hearing loss. The Company looks forward to initiating a Phase 1/2...

Privately-held NeuExcell Therapeutics has collaborated with Spark Therapeutics to develop a treatment for Huntington's Disease (HD). Spark Therapeutics is a part of Roche Holdings AG (OTC: RHHBY). Under the deal, Spark Therapeutics has the option to license...

​​​​​​BioMarin Pharmaceutical Inc (NASDAQ: BMRN) faces another setback in its gene therapy development program after the FDA placed the phenylketonuria (PKU) gene therapy trial on hold. The Phase 1/2 Phearless study is evaluating BMN 307, an AAV5-phenylalanine hydroxylase (PAH) gene...

Astellas Pharma Inc (OTC: ALPMF) has voluntarily paused screening and dosing of additional participants in its ASPIRO trial evaluating AT132 gene therapy in patients with X-linked Myotubular Myopathy (XLMTM). The decision follows a serious adverse event (SAE) in a study participant...

The European Commission has granted Orphan Drug designation to Taysha Gene Therapies Inc's (NASDAQ: TSHA) TSHA-105, AAV9-based gene therapy for SLC13A5-related epilepsy. Related: Taysha Gene Secures $100M In Debt Financing For Support Pipeline Development SLC13A5...

The FDA has lifted the clinical hold on Rocket Pharmaceuticals Inc's (NASDAQ: RCKT) Phase 1 trial of RP-A501 (gene therapy) for Danon Disease, allowing patient enrollment to resume.  The hold was removed after the Company addressed the FDA's requests to modify the...

The European Medicines Agency (EMA) Committee for Orphan Medicinal Products has issued a positive opinion on Akouos Inc's (NASDAQ: AKUS) application for orphan drug designation for AK-OTOF. AK-OTOF is a gene therapy intended for the treatment of otoferlin gene-mediated...

Shares for Bluebird Bio Inc (NASDAQ: BLUE) is hovering at a 52-week low after the FDA placed a clinical hold on clinical studies of elivaldogene autotemcel (eli-cel, Lenti-D) gene therapy for cerebral adrenoleukodystrophy (CALD). The company reported an adverse reaction to...

The European Medicines Agency has granted Priority Medicines designation to Mustang Bio Inc's (NASDAQ: MBIO) MB-107 for X-linked severe combined immunodeficiency (X-SCID) in newly diagnosed infants, also known as bubble boy disease. The Company is preparing to initiate a...

Abeona Therapeutics Inc (NASDAQ: ABEO) has completed a Type B meeting with the FDA regarding the pivotal trial for its ABO-102 gene therapy for Sanfilippo syndrome type A (MPS IIIA).  ABO-102 is currently being evaluated in the single-arm Transpher A study in patients with MPS...