After gaining U.S. FDA priority approval for the first gene therapy to treat hemophilia B, CSL Ltd. reported long-term data from the pivotal HOPE-B trial that showed a single infusion of Hemgenix (etranacogene dezaparvovec-drlb) generated elevated and sustained mean factor IX levels and reduced the rate of annual bleeding. Presented at the American Society of Hematology (ASH) annual meeting on Dec. 10, data showed 24-month results reinforced the safety of treatment, with no serious treatment-related adverse effects.
The U.S. FDA gave its go-ahead for Hemgenix (etranacogene dezaparvovec-drlb), Uniqure NV’s one-time gene therapy – the first for the treatment of adults 18 and older living with hemophilia B. Patients have been waiting “maybe beyond two decades” for a new therapy, Uniqure CEO Matthew Kapusta said. Hemgenix emerged from pioneering work by St. Jude Children’s Research Hospital and the University College London.
By pairing the expression of an inhibitory ion channel with an activity-dependent promoter, researchers have developed the first on-demand gene therapy that specifically silenced hyperactive cells and prevented epileptic seizures.
Abeona Therapeutics Inc. is on the road to filing a BLA with the U.S. FDA after posting positive top-line phase III data in wound healing and also to the bank with a new $35 million private placement financing. The data for EB-101, an autologous cell therapy, came from a pivotal study of treating recessive dystrophic epidermolysis bullosa, an ultra-rare connective tissue disorder. Results showed the study met its two co-primary endpoints in wound healing and for reducing pains in large, chronic wounds caused by the disorder.
“Big genomics” specialist Replay Holdings LLC has unveiled the first of four satellite genomic medicine companies it is forming to apply its high capacity herpes simplex viral (HSV) vector to next generation gene therapies. Eudora Therapeutics will specialize in inherited retinal eye diseases. It arrives on the scene with programs targeted at retinitis pigmentosa, Stargardt disease and Usher syndrome type 1B.
Astellas Pharma Inc. has invested $50 million in Taysha Gene Therapies Inc. in exchange for 15% of the company and exclusive options to in-license Taysha’s lead gene therapy candidates, TSHA-102 for Rett syndrome and TSHA-120 for giant axonal neuropathy.
The extraordinary proliferation of different genetic therapeutic modalities in the last decade has not been matched by a commensurate flourishing of delivery technologies. The liver is the natural destination for many carriers when administered systemically. But getting beyond the liver to other organ systems has proven to be a significant challenge. Only a tiny percentage of carriers – even those with targeting moieties – escape first pass metabolism in the liver and reach their target destination. “The key issue is getting out of the liver,” Nessan Bermingham, founder and interim CEO of Liberate Bio, Inc., told BioWorld. “That’s not a trivial problem.”
The volatile gene therapy space is getting a boost with Eli Lilly and Co.’s acquisition of Akouos Inc., which only a month before had received the first IND from the U.S. FDA for an adeno-associated virus (AAV)-based hearing loss treatment. Lilly plans to pay about $610 million for the company to get at AK-OTOF, Akouos’ lead candidate for treating hearing loss due to mutations in the otoferlin gene.
Bloomsbury Genetic Therapies Ltd. has raised £5 million (US$5.5 million) in a seed round, to take four gene therapy programs based on research carried out by the scientific founders at University College London into clinical development.
After winning the backing of European regulators, Biomarin Pharmaceutical Inc. is returning to the U.S. FDA with its hemophilia A gene therapy, valoctocogene roxaparvovec, following a rejection in August 2020. It's armed with data it hopes will assuage concerns about long-term safety and benefits.
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