gene therapy

Adverum Biotechnologies Inc (NASDAQ: ADVM) announced the presentation of a new post-hoc analysis from the OPTIC study of ADVM-022 in wet age-related macular degeneration (wet AMD). Data were shared at the Association for Research in Vision and Ophthalmology (ARVO) 2022 Annual...

After the FDA lifted its hold on a late-stage study, Pfizer Inc (NYSE: PFE) said it would open the first U.S. trial sites for its experimental gene therapy for a muscle-wasting disorder. The FDA had put Pfizer's Investigational New Drug (IND) application for fordadistrogene...

The independent Data and Safety Monitoring Board (DSMB) for Ocugen Inc's (NASDAQ: OCGN) Phase 1/2 trial of OCU400 has recommended that the study proceeds with enrolling additional subjects. The recommendation follows the review of safety data based on dosing to date. OCU400 is...

Amicus Therapeutics Inc (NASDAQ: FOLD) has announced additional results from a Phase 1/2 clinical study of AT-GAA in adult patients with Pompe disease. Study participants treated with AT-GAA for up to 36 months showed persistent and durable effects on six-minute walk test (6MWT)...

The FDA has outlined new draft guidance on what should be submitted in a clinical trial application and potentially concerning safety issues to track the genome editing products. The guidance recommends at least 15 years of long-term follow-up after product administration, aligning with...

Hit by several delays for its gene therapy programs, Bluebird bio Inc (NASDAQ: BLUE) ended 2021 with a cash and equivalents balance of approximately $442 million. The full-year 2022 cash burn is expected to be less than $400 million.  The company's expectation of generating...

Amicus Therapeutics Inc (NASDAQ: FOLD) has terminated its plans to spin off its gene therapy assets, citing 'existing market conditions.' With "market conditions" sinking the planned merger with ARYA Sciences Acquisition Corp IV (NASDAQ: ARYD), Amicus plans to...

Takeda Pharmaceutical Co Ltd (NYSE: TAK) has signed up for a gene therapy pact, encoding a $2 billion biobucks deal with Code Biotherapeutics across four programs. The collaboration entails deploying Code's non-viral gene therapies against liver-directed rare disease...

BioMarin Pharmaceutical Inc (NASDAQ: BMRN) said that the FDA had requested additional information regarding the clinical hold on PHEARLESS Phase 1/2 study of BMN 307 issued in September 2021.  BMN 307 is an AAV5-human phenylalanine hydroxylase (hPAH) gene therapy studied in...

Decibel Therapeutics Inc (NASDAQ: DBTX) announced the presentation of preclinical data on DB-OTO, its lead gene therapy product candidate. DB-OTO is a dual-vector adeno-associated candidate designed to restore hearing to individuals with profound, congenital hearing loss caused by mutations...

Regenxbio Inc (NASDAQ: RGNX) announced additional interim data from Cohorts 1-3 of Phase 1/2 trial of RGX-121 for patients up to 5 years old diagnosed with Mucopolysaccharidosis Type II (MPS II),  The data were shared at the 18th Annual WORLDSymposium.   RGX-121 is an...

Avrobio Inc's (NASDAQ: AVRO) gene therapy for a rare lysosomal storage disorder showed potential durability in the first three patients more than one year after infusion. The data comes from a Phase 1/2 trial testing the safety and efficacy of AVR-RD-04 for cystinosis who had...

Sangamo Therapeutics Inc (NASDAQ: SGMO) announced updated preliminary results from the Phase 1/2 STAAR study of isaralgagene civaparvovec (ST-920), Fabry disease gene therapy candidate. Fabry disease is an inherited disorder that results from the buildup of a particular type of fat...

Sio Gene Therapies Inc (NASDAQ: SIOX) has decided to terminate its licensing agreement with Oxford Biomedica for AXO-Lenti-PD, its lentiviral gene therapy program for Parkinson's disease. Due to several factors, the Company is deprioritizing its Parkinson's disease...

Taysha Gene Therapies Inc (NASDAQ: TSHA) has reported clinical efficacy and safety data for the high dose cohort of 3.5x1014 total vg and long-term durability data across all therapeutic doses of TSHA-120 in giant axonal neuropathy (GAN). 5-point improvement was observed in the rate...