Tessera Therapeutics Inc.’s lead in vivo gene editing program, TSRA-196, has been awarded orphan drug and fast track designations by the FDA for adults with α-1 antitrypsin deficiency (AATD).

Precision Biosciences Inc. has received FDA clearance of its IND application enabling clinical trial site activation for a phase I/II trial (FUNCTION-DMD) of PBGENE-DMD for the treatment of ambulatory patients with Duchenne muscular dystrophy (DMD). The first site in the U.S. will be activated in the first half of this year.

Seamless Therapeutics has received big pharma endorsement of its proprietary recombinase gene editing platform, sealing a potential $1.12 billion deal with Eli Lilly and Co. to apply the technology in hearing loss.

Seamless Therapeutics has received big pharma endorsement of its proprietary recombinase gene editing platform, sealing a potential $1.12 billion deal with Eli Lilly and Co. to apply the technology in hearing loss.

Following a clinical hold last October of Intellia Therapeutics Inc.’s Magnitude and Magnitude-2 phase III trials of CRISPR/Cas9 gene editing therapy nexiguran ziclumeran (nex-z) to treat transthyretin amyloidosis with cardiomyopathy (ATTR-CM) and polyneuropathy (ATTR-PN), respectively, the U.S. FDA lifted the hold on Magnitude-2, pushing the company’s shares up by 22% in early trading Jan. 27.

There was an upbeat message for cell and gene therapy companies in the 2026 industry update presented as the J.P. Morgan Healthcare Conference opened on Monday, with Tim Hunt, CEO of the Alliance for Regenerative Medicine, telling delegates that after lean years of learning, adapting and setbacks, the sector is now self-sustaining.

Gene editing can repair mutations that prematurely halt protein synthesis, resulting in incomplete peptides that cause various diseases. However, other approaches achieve the same effect without altering the genome. Startup Alltrna Inc. has developed a strategy based on transfer RNA to bypass the premature stop codons that end early protein translation. The company already has a first clinical candidate that could treat metabolic diseases such as methylmalonemia or phenylketonuria.

Gene editing can repair mutations that prematurely halt protein synthesis, resulting in incomplete peptides that cause various diseases. However, other approaches achieve the same effect without altering the genome. Startup Alltrna Inc. has developed a strategy based on transfer RNA (tRNA) to bypass the premature stop codons that end early protein translation. The company already has a first clinical candidate that could treat metabolic diseases such as methylmalonemia (MMA) or phenylketonuria (PKU).

Regeneron Pharmaceuticals Inc. and Tessera Therapeutics Inc. have established a global collaboration to develop and commercialize TSRA-196, Tessera’s lead investigational in vivo gene editing program for the treatment of α-1 antitrypsin deficiency (AATD).

San Francisco Bay Area researchers from UC Berkeley, UC San Francisco and Stanford University have combined their technologies to create Azalea Therapeutics Inc., a company focused on editing cells in vivo.