Gene therapy has had its commercial struggles in the past year. The cost to patients is in the millions and fewer are stepping forward for treatment than companies would like. While development continues in this game-changing field, some have struggled with regulatory authorities during development while others have just stepped away altogether.
Rznomics Inc. continued South Korea’s year-end biotech rally with a ₩46.35 billion (US$31.35 million) IPO Dec. 18. Proceeds will fund Seongnam-si, South Korea-based Rznomic’s pipeline of gene therapies, built on the company’s trans-splicing ribozyme RNA Replacement Enzyme technology platform.
Addition Therapeutics came out of stealth mode to highlight its Precise RNA-Mediated Insertion of Transgenes (PRINT) gene therapy platform. The system is based on research of retrotransposons by Kathleen Collins’ laboratory at University of California, Berkeley, that was spun out into Addition in 2021.
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.
Only a couple of years since the first sickle cell disease (SCD) gene therapies gained U.S. FDA approval, researchers are working to expand access for younger children, and to improve manufacturing and commercialization to reach patients faster.
More than six years after Novartis AG’s Zolgensma was approved for children under 2 with spinal muscular atrophy (SMA) with biallelic mutations in the survival motor neuron 1 gene, the U.S. FDA cleared a new version, under the brand name Itvisma (onasemnogene abeparvovec), for those 2 and older, including teens and adults with the same mutation.
Korro Bio Inc.’s latest update on RNA editing prospect KRRO-110 may mean one less competitor in alpha-1 antitrypsin deficiency (AATD), and shares of the firm (NASDAQ:KRRO) closed Nov. 13 at $6.50, down $24.92, or 79%. As part of the third-quarter earnings report, Korro said KRRO-110 produced functional protein in AATD patients but fell short of projected levels of functional protein after a single administration.
Engene Holdings Inc.’s protocol amendment to its phase II trial with detalimogene voraplasmid in bladder cancer worked out in a big way, and shares of the firm closed Nov. 11 at $8.82, up $2.81, or 47%. Engene rolled out additional preliminary data from the pivotal cohort of the ongoing Legend study testing the nonviral gene therapy in high-risk, Bacillus Calmette-Guérin-unresponsive non-muscle invasive bladder cancer with carcinoma in situ, with or without concomitant papillary disease.
The U.K. Medicines and Healthcare products Agency (MHRA) has committed to major reforms of how it regulates drugs for rare diseases, making it easier to run clinical trials and get approvals. The new rules will be published in full early in 2026 and come into effect later in the year, but following consultation with industry, academics and patients’ groups, the agency has released a position paper setting out its plans.
RSS
