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The FDA turned away Ebvallo in January, taking issue with the design of the registrational trial. In a recent meeting, however, the agency agreed that the study could in fact support the cell therapy’s approval. The news comes a week after the departure of controversial biologics Director Vinay Prasad.
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The Department of Health and Human Services is spinning its wheels, unable to establish steady leadership at three major divisions—the CDC and the FDA’s two primary review units.
Robert F. Kennedy Jr.’s health department has consistently touted radical transparency as being key to its mission. Recent instances—the FDA’s decision not to disclose the recipients of three Commissioner’s National Priority Vouchers and FDA and CDC choices not to publish vaccine-related papers—call this intent into question.
In Salt Lake City, biotech founders new and seasoned reflect on ways to ride out the industry’s challenges, such as sending cold emails to investors and learning to address leadership weaknesses.
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Psychedelics are a “game changer” in depression care, according to William Blair, but the complicated treatment regimens mean they will likely be supplanted by more-traditional options once they become available.
The alliance, which pairs Tenaya’s modality agnostic target identification and validation capabilities with Alnylam’s deep experience in RNA interference therapeutics, comes during a period of resurgence for the cardiovascular space.
UniQure’s Path for Huntington’s Gene Therapy Clouded by Ethical Questions as Potential Phase 3 Looms
While the FDA appears to be adamant that uniQure conduct a sham surgery–controlled Phase 3 trial before AMT-130 can be considered for approval, experts believe there is an alternate path forward for the therapy, perhaps even based on precedent from the recent drama surrounding Moderna’s mRNA flu vaccine.
Adam Urato, who is currently a vaccine advisor to the CDC, is closely associated with acting CDER director Tracy Beth Høeg and is a fellow skeptic of the use of selective serotonin reuptake inhibitors during pregnancy.
The timing of the partial hold is “odd,” according to analysts at Stifel, who noted that the preclinical data the FDA took issue with were filed in mid-2024.
South Korea has attracted increasing investment from the pharmaceutical industry, which is drawn to the Asian country due to its experience in antibody-drug conjugates and cell and gene therapies, according to McKinsey.
In this episode of Denatured, you’ll listen to Ram May-Ron, managing partner at FreeMind Group, and Ravi Kiron, managing director at Biopharma Strategy Advisors. We’ll be speaking about how to combine nondilutive funding and family office money into a unified strategy that gets companies through the drug development valley of death.
Over half of biopharma professionals would work again at the companies that let them go, according to a BioSpace LinkedIn poll. Several professionals, as well as recruiting and talent acquisition experts, discuss reasons for—and a key risk of—going back.
Having targeted Hims & Hers last year, the FDA has issued warnings to more telehealth companies over their promotion of GLP-1 drugs used to treat diabetes and obesity. In one case, compounded products were linked with multiple ER visits.
UniQure and REGENXBIO are both dealing with FDA setbacks for their respective gene therapies, as regulatory experts question the FDA’s decision-making processes; CBER director Vinay Prasad is under probe for allegedly fostering a toxic workplace; Sarepta CEO Doug Ingram is stepping down after several years of tumult at the top of the muscular dystrophy–focused company; and Eli Lilly again tops Novo Nordisk in a weight loss trial.