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.

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).

Emulate Inc. has launched a new adeno-associated virus (AAV) transduction application for the Liver-Chip that enables gene therapy researchers to test the delivery efficiency and safety of AAV vectors in a validated, human-relevant model of the liver and get results in weeks.

Although gene therapy is now “a clinical reality,” it still remains an early stage therapeutic modality. That’s the view of Caroline Man Xu, CEO and co-founder of Vigeneron GmbH, a German gene therapy company that has maintained a low profile while steadily staking out a promising position in gene therapies for inherited retinal disease.

Novartis AG, an early and active player in bringing gene therapies to market, has agreed to pay Voyager Therapeutics Inc. $54 million up front and up to $1.7 billion in fees and milestone payments for options to license up to five next-generation adeno-associated virus (AAV) capsids to use as gene therapy vectors for neurological diseases.

Japan’s Astellas Pharma Inc. is continuing its investment in gene therapies, following up its $3 billion acquisition of Audentes Therapeutics Inc. with a technology licensing deal with Dyno Therapeutics Inc. potentially worth more than $1.6 billion. Central to the deal is Cambridge, Mass.-based Dyno’s adeno-associated virus (AAV) vector technology, which can be used to direct gene therapies to skeletal and cardiac muscle.

Delivering antibodies in the form of their DNA could enable their therapeutic use under several circumstances where traditional antibodies fall short. One of those is resource-poor settings where the current cost of antibodies makes them a nonstarter. Perhaps the largest opportunity to expand antibody use in such settings is for HIV, where broadly neutralizing antibodies have the potential to be the next best thing to a vaccine or a cure – if they can be made to last, for cheap.

Monoclonal antibodies are a triumph of modern medicine. They are also too expensive to be a standard therapy in all but the wealthiest countries. “Having 10% or 15% of your population on antibodies is not sustainable even in wealthy countries,” Rachel Liberatore told BioWorld. Liberatore is director of research and development at Renbio Inc., which is testing the intramuscular delivery of antibody-encoding DNA to prevent and treat infections, including SARS-CoV-2 and HIV.

Researchers at Oregon Health and Science University have used mouse models to estimate the frequency at which gene therapies delivered by adeno-associated virus (AAV) vectors integrated into host DNA, and come up with an estimate of up to roughly 3% – a number that is orders of magnitude higher than previous estimates and would translate into several hundred million cells with integrated viral vectors in an adult liver, assuming that 10% of cells took up the transgene.