Using informed consent to do what Congress couldn’t, the U.S. FDA is flexing its regulatory authority to halt clinical trials that involve sending cells from American patients to China or other adversarial nations for genetic engineering and subsequent infusion back into the patient.
The realignment within the U.S. FDA continued with reports of the removal of two high level executives. When asked by BioWorld if the Center for Biologics Evaluation and Research’s (CBER) Office of Therapeutic Products director and deputy director had been forced out and if so, why, an HHS spokesperson responded on background with a single sentence: “Center directors deserve to be supported by managers that are aligned with aggressive goals to expeditiously advance therapeutics for rare diseases using the gold standard of science.”
With plenty of GLP-1 money to spend, Eli Lilly and Co. is buying Verve Therapeutics Inc. and its gene-editing program for about $1.3 billion. Two of Verve’s one-time treatments are in the clinic. Lead candidate VERVE-102, a gene-editing treatment targeting PCSK9, is in a phase Ib study to reduce cholesterol levels.
How the U.S. FDA might respond became a serious question for Wall Street as Sarepta Therapeutics Inc. made known a second death due to acute liver failure with gene therapy Elevidys (delandistrogene moxeparvovec), cleared for Duchenne muscular dystrophy (DMD). Shares of Cambridge, Mass.-based Sarepta (NASDAQ:SRPT) closed June 16 at $20.94, down $15.24, or 42%, as Wall Street digested the news.
Capsida Biotherapeutics Inc. has obtained IND clearance from the FDA for CAP-003, an intravenously administered gene therapy, for Parkinson’s disease associated with GBA mutations (PD-GBA). A phase I/II trial will begin dosing in the third quarter of this year.
Three months after dosing the first patient with its dual vector gene therapy, Splicebio SL has closed a $135 million series B to fund the phase I/II trial of SB-007 in the treatment of Stargardt’s disease to completion. Other adeno-associated viru gene therapies for the inherited retinal disorder have entered the clinic, but SB-007 is the first with the capacity to deliver a full version of the ABCA4 gene that underlies Stargardt’s.
Children with solid tumors who relapse are being treated with the same chemotherapy they would have been given 40 years ago, as “there have been no major approvals for pediatric solid tumors,” Catherine Bollard, senior vice president and chief research officer at Children’s National Hospital, said at a June 5 FDA roundtable on cell and gene therapies (CGTs). The problem isn’t the science. Bollard said many groups are working on curative CGTs “for these children who have lost all other hope for survival.” The real gap is that “big pharma doesn’t see the business model because it’s a rare disease,” she added.
Regenxbio Inc.’s gene therapy in treating Duchenne muscular dystrophy (DMD) produced positive initial phase I/II results from its first five patients. However, the company’s stock (NASDAQ:RGNX) shuddered on June 5 as shares closed at $8.36 each, a drop of 17% on the day.
Chronic pain is a constant challenge to around 20% of the global population, and treatments to mitigate such pain often cause unacceptable side effects because the receptors and signaling pathways involved in pain sensing also drive necessary processes in the heart, lungs and liver. Opioid analgesics can be effective against chronic pain, but they can lead to tolerance and addiction.
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