Cell and Gene Therapy
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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.
Novo Nordisk pulled back from cell therapies last October, scrapping development of a type 1 diabetes therapy and laying off most employees working on this modality.
The arrangement will boost AstraZeneca’s cell therapy portfolio as the pharma targets $80 billion in revenue by 2030.
2026 is shaping up to be a pivotal year for rare disease drugmakers, with key approvals, filings and readouts lined up for this year.
AstraZeneca is relying on several upcoming products to help hit its target of $80 billion in revenue by 2030, including drugs for hypertension, breast cancer and generalized myasthenia gravis, all of which are currently under FDA review.
Jefferies analysts forecast a $1 billion market opportunity for each of Sarepta’s siRNA programs for facioscapulohumeral muscular dystrophy and myotonic dystrophy type 1.