Featured Story | By Fraiser Kansteiner Cell and gene therapies are already revolutionizing treatment for some diseases, but scores more are working their way toward market—and they all have to make it through the tricky step of scaling up manufacturing. Some players are reimagining how it's done. read more |
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Top Stories By Amirah Al Idrus Caribou Biosciences is the latest biotech to rake in an upsized IPO. The cell therapy player bagged $304 million in a Wall Street debut, which will see its lead CAR-T program through a first phase 1 readout and propel two prospects toward—and maybe also into—the clinic. read more By Amirah Al Idrus Six weeks after landing in the clinic, Bayer’s stem cell therapy for Parkinson’s disease is getting into the fast lane. The German pharma picked up the treatment, DA01, by buying Versant Ventures out of their regenerative medicine joint venture BlueRock Therapeutics in a deal worth about $1 billion. read more By Annalee Armstrong Biogen had two high profile gene therapy failures in the middle of the Aduhelm craze, and now they’re going to pay for it. The company has reported $542 million in impairment charges over the two trial flops during second quarter earnings. read more By Amirah Al Idrus When Dan Kemp was plotting his next move after Takeda, he was “blown away” by data from Wugen, a biotech working on off-the-shelf natural killer cell therapies. Now, after four months in the CEO seat, he’s ready to take those treatments to the next level with a $172 million financing. read more By Arlene Weintraub Scientists from Ohio State University developed a novel method for delivering gene therapy to specific regions of the brain and tested it in children with a rare neurodegenerative disorder called AADC deficiency. Based on positive results from the trial, they're now eyeing the technology in more widespread brain disorders like Alzheimer's. read more By Ben Adams Tenaya Therapeutics is fresh off a meaty raise but is quickstepping into a $100 million IPO attempt. read more By Amirah Al Idrus Kriya Therapeutics is looking beyond the bread and butter of gene therapy—rare diseases—to instead focus on programs for more complex and common diseases like diabetes. And now, the Redwood City, California-based company has banked another $100 million to support the work. The new funds will boost Kriya's technology and expand its pipeline, as well as propel assets in oncology, eye diseases and metabolic diseases forward. read more By Amirah Al Idrus Sigilon’s encapsulated cell therapy for hemophilia A has hit a regulatory snag. The FDA paused the phase 1/2 study after a patient developed a serious side effect that caused their body to attack the treatment. The hold comes after Sigilon alerted the agency and other regulators to this issue and a temporary halt to enrollment in the trial. read more By Nick Paul Taylor And just like that, half of Adverum Biotechnologies' pipeline is gone. The biotech announced after market Thursday that it would abandon a key eye disease indication and shift focus to another, but analysts aren't sure how the company will overcome the adverse events that sent them in the new, slimmed-down direction. read more By Amirah Al Idrus As Lexeo Therapeutics ushers two gene therapies toward the clinic, the company is adding another three heart disease programs to its already busy pipeline. The assets come through Lexeo’s acquisition of Stelios Therapeutics, which was founded on research out of the University of California, San Diego. read more By Nick Paul Taylor Coave Therapeutics has added €21.2 million ($25.0 million) to its series B round, setting the French biotech up to move a gene therapy against PDE6b inherited retinal dystrophies toward pivotal trials. read more By Angus Liu Scientists at Texas Heart Institute have developed a gene therapy to repair heart damage after a heart attack and reported positive outcomes in pigs. It involves a DNA-silencing RNA molecule that suppresses the Hippo signaling pathway. In pig models of heart attack, the therapy improved heart function, the researchers reported. read more Resources Sponsored by: Box Read "Get to Know Cloud Content Management for Life Sciences" and learn how to eliminate silos across all workflows, from R&D to commercialization. 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