Children and adults with a type of congenital hearing loss now have a free treatment option, with the U.S. FDA’s accelerated approval of Regeneron Pharmaceuticals Inc.’s DB-OTO, an AAV-mediated gene therapy. Branded Otarmeni (lunsotogene parvec), it is cleared specifically for hearing loss caused by variants in the otoferlin gene.

The already-thriving CAR T space took another big stride forward as Eli Lilly and Co. disclosed its plan to acquire Boston-based Kelonia Therapeutics Inc. for as much as $7 billion in cash, including an up-front payment of $3.25 billion, with the rest coming if clinical, regulatory and commercial goals are reached.

Roche Holding AG is making good on its promise to try and convince the EMA of the benefits of Elevidys (delandistrogene moxeparvovec), announcing a further global phase III trial of the Duchenne muscular dystrophy gene therapy.

The U.S. FDA’s latest draft guidance on gene therapies focuses on nonclinical studies using next-generation sequencing-based methods and bioinformatics to evaluate safety risks associated with off-target editing and loss of genome integrity in human gene-edited products.

“Home-run” efficacy and what Cowen analyst Tyler Van Buren called “pristine” safety in the phase II Alpha3 study with CAR T therapy cemacabtagene ansegedleucel (cema-cel) sent shares of Allogene Therapeutics Inc. (NASDAQ:ALLO) skyward to a $4.46 high, up nearly 64%, on April 13. Shares eventually closed at $3.06, up 34 cents.

Chiesi Group’s idebenone faced a regulatory setback last month after the U.S. FDA issued a complete response letter (CRL) to the company’s NDA for Leber hereditary optic neuropathy (LHON), a rare inherited disorder that causes sudden vision loss.But a chance missed for Chiesi may be an opportunity for gene therapies, including Gensight Biologics SA’s lenadogene nolparvovec (Lumevoq; GS-010).

In a win for the rare disease space, the U.S. FDA granted accelerated approval for Rocket Pharmaceuticals Inc.’s Kresladi (marnetegragene autotemcel) as the first gene therapy option for treating severe leukocyte adhesion deficiency-I (LAD-I), an ultrarare genetic immune disorder characterized by an immunodeficiency predisposing those affected to recurrent and fatal infections.

With top-line pivotal data with gene therapy RGX-202 for Duchenne muscular dystrophy (DMD) due in the next quarter, Regenxbio Inc. rolled out positive interim data from the phase I/II Affinity trial at the Muscular Dystrophy Association Clinical and Scientific Conference (MDA) in Orlando, Fla., where Bridgebio Pharma Inc., Capricor Therapeutics Inc., and Solid Biosciences Inc. also had clinical findings to talk about.

The regulatory clouds that have been darkening the U.S. FDA landscape of late for Uniqure NV’s gene therapy AMT-130 in Huntington’s disease may be parting a bit with the announced departure of Vinay Prasad as director of the agency’s CBER at the end of April.

Entering its first major cardiovascular disease collaboration with a biopharma company, while it advances two internal gene therapies, Tenaya Therapeutics Inc. signed on with Alnylam Pharmaceuticals Inc. to deliver up to 15 novel genetic targets that could lead to new heart disease medicines. The deal comes with $10 million up front, and up to $1.13 billion is available to South San Francisco-based Tenaya if all targets meet certain milestones, leading to approved therapeutics that Alnylam develops and commercializes.