Little more than a month after the U.S. FDA approved the first gene therapy for adults with hemophilia B, Uniqure NV’s Hemgenix, strong phase III data have come from Pfizer Inc. The Pfizer results show fidanacogene elaparvovec, a vector containing an AAV capsid and a high-activity human coagulation factor IX (FIX) gene for treating adult men with moderately severe to severe hemophilia B, hit the primary endpoint in the phase III Benegene-2 study. The one-time therapy is designed to allow those living with hemophilia B to be able to produce FIX instead of receiving regular, ongoing doses of exogenous FIX.

CRISPR gene editing has been one of the important advances of the last decade, in biotechnology and increasingly in medicine. First applied to human cells in 2013, and honored with the 2020 Nobel Prize in Physiology or Medicine, its meteoric rise can make CRISPR look like the molecular equivalent of a miracle healer. But in the research and clinical trenches, CRISPR-based approaches, like any others, need to find applications where their desired effects outweigh their side effects. And finding those applications necessitates ways to identify off-target effects.

Neurophth Therapeutics Inc. has received FDA clearance of its IND application for the in vivo gene replacement therapy NFS-02, a novel recombinant adeno-associated viral serotype 2 vector (rAAV2) containing a codon-optimized NADH-dehydrogenase subunit 1 (ND1) gene, for the treatment of Leber hereditary optic neuropathy (LHON) associated with ND1 mutation.

Ferring Pharmaceuticals A/S has notched another U.S. FDA approval, this time for a bladder cancer treatment, Adstiladrin (nadofaragene firadenovec). The non-replicating adenovirus vector-based gene therapy’s approval comes only weeks after the FDA’s Nov. 30 approval of the privately held company’s Rebyota (fecal microbiota, live), the first fecal microbiota treatment in the U.S. Adstiladrin is another landmark, as the first FDA-approved gene therapy to treat high-risk, non-muscle-invasive bladder cancer. Saint-Prex, Switzerland-based Ferring said it anticipates the product becoming commercially available in the U.S. in the second half of 2023.

Eikonoklastes Therapeutics Inc. and Forge Biologics Inc. have established a manufacturing partnership to advance Eikonoklastes' adeno-associated viral (AAV)-based gene therapy, ET-101, into clinical trials for the treatment of patients with amyotrophic lateral sclerosis (ALS).

The FDA has awarded orphan drug designation to Tenaya Therapeutics Inc.'s gene therapy product candidate, TN-401, for the treatment of arrhythmogenic right ventricular cardiomyopathy (ARVC). TN-401 is an adeno-associated virus (AAV)-based gene therapy being developed for the treatment of genetic ARVC caused by plakophilin-2 (PKP2) gene mutations.