Gene therapy has finally become the “new normal” with serial breakthroughs unlocking “tremendous value” for patients and society, while at the same time the U.S. health care system is shaping up to enable access to these costly treatments, according to Tim Hunt, CEO of the Alliance for Regenerative Medicine. The capital markets were causing grief, but 2023 saw the field maturing, with seven FDA and one EMA approval. The prospect is that in 2024, five gene therapies for rare genetic diseases will be approved, along with the first-ever adoptive cell therapy for treating solid tumors and the first allogeneic T-cell therapy.

In California: R&D delay grounds for class action

A California state appeals court yesterday opened an avenue, in California at least, for class action lawsuits against drug and device companies based on their pipeline development priorities. In this instance, the court said a suit could continue against Gilead Sciences Inc., which the plaintiffs alleged was negligent in delaying development of a second-generation HIV drug that carried less risk than the company’s first-generation drug, tenofovir alafenamide fumarate. While the court is letting the case proceed on the negligence question based on California law, it didn’t say Gilead should have brought the second version to market sooner, Evercore ISI analyst Umer Raffat noted. “Bottom-line: this is not the outcome Gilead would have hoped for … but it’s also not the worst-case outcome,” Raffat said.

Relay making strides in FGRF2, PI3Ka

Relay Therapeutics Inc. was among the firms to update on progress at the J.P. Morgan Healthcare Conference in San Francisco. The company has PI3Ka and FGRF2 in the works. Both emerged from Cambridge, Mass.- based Relay's Dynamo platform.

WLF sees First Amendment issues in US FDA draft off-label guidance

The U.S. FDA’s latest draft guidance on discussions of off-label uses with doctors revisits a controversial subject that has previously migrated into the courts, and by some accounts, may do so yet again. In comments to the docket, the Washington Legal Foundation (WLF) argued that this latest attempt to regulate commercial speech is another example of the agency’s “flagrant disregard for drug and device manufacturers’ free speech rights,” which WLF seemed to suggest is an actionable violation of the First Amendment.

MS? Blame great-great-great-great-great-great-great-great-grandma

Current risk genes for some diseases such as multiple sclerosis may have emerged in the past as protection against infection by different pathogens. A group of researchers led by scientists from the University of Copenhagen has analyzed the ancient DNA of European populations and has revealed how MS, Alzheimer’s disease and diabetes arose as populations migrated. This evolution would explain the modern genetic diversity and the incidences of these pathologies observed today in the old continent.

Asia CDMO giants Wuxi and Samsung to grow production, ADC capacity

Major contract research development and manufacturing organizations (CDMO) out of Asia are announcing plans to ramp up production and antibody-drug conjugate (ADC) capabilities worldwide. China’s Wuxi Biologics Inc. said on Jan 8, ahead of a presentation at the 2024 annual J.P. Morgan (JPM) Healthcare Conference in San Francisco, that it would ramp up U.S. production. South Korean CDMOs Samsung Biologics Co. Ltd. and Lotte Biologics Co. Ltd. also unveiled hiked up plans for production. On the agenda of all three CDMOs were efforts to scale ADC capabilities, either independently or through partners.

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