gene therapy

Taysha Gene Therapies Inc (NASDAQ: TSHA) reported initial serum β-hexosaminidase A (Hex A) enzyme activity data for TSHA-101 in patients with Sandhoff and Tay-Sachs diseases, two forms of GM2 gangliosidosis.  According to Taysha, TSHA-101 is the first bicistronic vector in...

The FDA has issued a hold, pending Chemistry, Manufacturing & Controls (CMC) clearance, on Mustang Bio Inc's (NASDAQ: MBIO) Investigational New Drug (IND) application for MB-207 in patients with X-linked severe combined immunodeficiency (XSCID). The application was submitted...

The FDA has extended the review period for Bluebird bio Inc's (NASDAQ: BLUE) biologics licensing applications (BLA) for its lentiviral vector gene therapies. The agency has pushed the applications for betibeglogene autotemcel (beti-cel) for β-thalassemia and...

Sarepta Therapeutics Inc (NASDAQ: SRPT) is walking away from a three-year licensing pact for a rare disease gene therapy candidate, leaving its former partner on its own as it prepares for a pivotal readout in mid-year. According to Lysogene, Sarepta returned the global commercial...

Bluebird bio Inc (NASDAQ: BLUE) announced planned updates to be presented at the 40th Annual J.P. Morgan Healthcare conference, including 2022 program milestones and financial outlook. In 2022, bluebird is focused on the FDA review of two gene therapies – betibeglogene autotemcel (beti-...

BioMarin Pharmaceutical Inc (NASDAQ: BMRN) announced results from its ongoing Phase 3 GENEr8-1 study of valoctocogene roxaparvovec, investigational gene therapy for severe hemophilia A.  Annualized Bleeding Rate (ABR) was significantly reduced by 4.1 treated bleeds per year in...

In a community letter, Pfizer Inc (NYSE: PFE) informed the Parent Project Muscular Dystrophy of the death of a young man in the Phase Ib trial of its mini-dystrophin gene therapy. The event has triggered a halt in screening and dosing and a clinical hold imposed by the FDA. Pfizer wrote...

Adverum Biotechnologies Inc (NASDAQ: ADVM) plans to conduct a Phase 2 trial of ADVM-022 in wet AMD at 2 X 10^11 vg/eye dose and a new lower 6 X 10^10 vg/eye dose, with three new enhanced steroid prophylaxis regimens in wet age-related macular degeneration (wet AMD).  The...

The FDA has placed a partial clinical hold on Bluebird bio Inc's (NASDAQ: BLUE) lovotibeglogene autotemcel (lovo-cel) gene therapy program for sickle cell disease (SCD) for patients under the age of 18.  The partial, temporary suspension relates to an ongoing...

The FDA has accepted for priority review Buebird bio Inc's (NASDAQ: BLUE) marketing application for elivaldogene autotemcel (eli-cel, Lenti-D) for cerebral adrenoleukodystrophy (CALD) in patients less than 18 years of age.  The agency set a Prescription Drug User Fee Act (...

UniQure NV (NASDAQ: QURE) announced initial observations on the first four patients enrolled in the lower-dose cohort of Phase 1/2 trial of AMT-130 for Huntington's disease.  AMT-130 was generally well tolerated at the lower dose of 6x1012 vector genomes (vg). There have...

Bluebird Bio Inc (NASDAQ: BLUE) presented new results for betibeglogene autotemcel (beti-cel), investigational gene therapy for β-thalassemia (beta-thal) who require regular red blood cell (RBC) transfusions. New data demonstrate that adult and pediatric β-thalassemia...

Bluebird bio Inc (NASDAQ: BLUE) announced updated results from its Phase 1/2 HGB-206 study of lovotibeglogene autotemcel (lovo-cel; formerly LentiGlobin for SCD, bb1111) gene therapy for sickle cell disease. The updated data included further analyses from its pivotal cohort, HGB-206...

The FDA has placed a clinical hold on Selecta Biosciences Inc's (NASDAQ: SELB) Phase 1/2 trial of SEL-302 (which consists of MMA-101 plus ImmTOR) for methylmalonic acidemia (MMA). MMA is a disorder in which the body cannot break down certain proteins and fats, resulting in...

Spark Therapeutics, a unit of Roche Holdings AG (OTC: RHHBY), has announced data from its Phase 1/2 trial of SPK-8011 in hemophilia A. At a median efficacy follow-up of 33.4 months, the study found that 16 of 18 study participants had sustained factor VIII (FVIII), which permitted prophylaxis...