Cell and Gene Therapy
THE LATEST
The SPAC agreement values PrimeGen US at $1.5 billion in equity and gives it capital to advance its pre-clinical triple-activated mesenchymal stem cell pipeline into the clinic.
After a series of deaths in patients taking Sarepta Therapeutics’ gene therapies, doubt has crept into investor sentiments around the long-time Wall Street darling, and patients may soon begin looking elsewhere.
Investor enthusiasm and evolving FDA pathways are accelerating rare disease drug development, with ultrarare conditions like MPS II moving into the spotlight.
Nearly 100 biotechs went public amid the industry’s IPO frenzy in 2021, driven by an influx of pandemic-driven investments. But many of those companies have little to show investors.
The alliance will add to Roche’s RNAi efforts, which include the hypertension candidate zilbesiran, partnered with Alnylam under a July 2023 agreement.
After years of contraction, investors see biotech reentering a growth cycle driven by scientific progress, asset quality and renewed conviction in oncology, obesity and neuroscience innovation.
AstraZeneca has risen as one of pharma’s most prolific investors in China, including a $630 million pledge last week for full rights to AbelZeta’s cell therapy for cancer.
The discovery of a tumor in a patient who received REGENXBIO’s gene therapy for Hurler syndrome prompted the FDA to place a hold on that program along with the company’s Hunter syndrome program, which is awaiting an FDA decision on or before Feb. 8.
Phacilitate’s annual event dawns as cell and gene therapies reach a new tipping point: the science has hit new heights just as regulatory and government policies spark momentum and frustration.
From opening new therapeutic mechanisms to repairing neuronal damage, investigational molecules from Ventyx Therapeutics, AC Immune, Gain Therapeutics and more could shape the future of Parkinson’s disease treatment.