Gene editing
The strategic initiative will extend Metagenomi’s cash runway into the fourth quarter of 2027. That same year, the company expects to generate initial Phase I data for its lead asset MGX-001 in hemophilia A.
Vinay Prasad, chief of the FDA’s Center for Biologics Evaluation and Research, is planning to publish a paper this month to outline his office’s thinking on accelerating gene editing reviews.
Intellia earlier this year reported a similar grade 4 liver enzyme elevation associated with the gene therapy nexiguran ziclumeran, though analysts at BMO Capital Markets at the time brushed it off as a “non-concern.”
Vertex Pharmaceuticals commits $45 million upfront to leverage Enlaza Therapeutics’ War-Lock platform to create drug conjugates and T cell engagers for autoimmune diseases and gentler conditioning for sickle cell/beta thalassemia gene-editing therapy Casgevy.
Jefferies analysts called the proxy filing, which is a standard disclosure after a merger agreement, “much more intriguing than normal” given the regulatory turmoil it revealed.
The pending deal was rumored overnight after a report from the Financial Times, spurring analysts to speculate that if true, the entire gene editing space would see a boost at the markets.
Gene therapies have ridden investor mania to huge valuations but commercialization challenges have pushed market caps to the floor. At a roundtable last week, FDA leaders promised faster approvals and broad support to the industry.
While an adverse event reported in Intellia’s gene therapy trial was a “non-concern” for analysts, it follows a handful of patient deaths in other trials for the modality and sent the company’s stock tumbling in pre-market trading.
Most of the 15 million children with a rare disease have no FDA-approved treatments available to them. And when it comes to the most-rare conditions, there isn’t even a pipeline.
Taking center stage at the American Society of Gene and Cell Therapy meeting was the first-ever reported case of a personalized in vivo CRISPR editing therapy, which substantially eased the symptom burden in an infant.
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