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.
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.
In a deal that could bring more than $2.1 billion in payments to Arbor Biotechnologies Inc., 90-year-old Chiesi Group gained exclusive and global rights to develop and commercialize ABO-101 for primary hyperoxaluria type 1, an ultra-rare disease caused by a mutation in the AGXT gene, as well as an option to go after a limited number of additional targets.
Sickle cell disease (SCD) and transfusion-dependent β-thalassemia (TDT) are severe monogenic blood disorders caused by mutations in the β-globin gene (HBB), resulting in abnormal or insufficient production of adult hemoglobin (HbA). Among emerging therapeutic approaches, the reactivation of fetal hemoglobin (HbF) represents one of the most promising strategies for both conditions.
Editas Medicine Inc. has nominated a lead in vivo development candidate, EDIT-401, a potential one-time therapy designed to significantly reduce LDL cholesterol (LDL-C) levels. The in vivo gene editing medicine is designed to treat hyperlipidemia by directly editing the LDLR gene to increase LDLR protein expression and reduce LDL-C levels.
Modi Ventures closed its second fund with total commitments of $88 million as it continues its mission to invest in companies at the intersection of artificial intelligence, biology and medical technology. The venture capital firm sees the future of health care as a convergence of these technologies.
