Cell and Gene Therapy

Leveraging recently proposed regulatory flexibilities, the gene-editing sector is working to cut R&D costs and unlock indications that were previously economically unviable, according to a new playbook published by the Alliance for Regenerative Medicine.

Caribou Biosciences entered the scene in 2011 and went public in 2021. Over the course of its existence, the biotech has received funding and support from a handful of Big Pharma players, including Novartis, AbbVie and Pfizer.

Viking Therapeutics rode strong obesity data before a major capital raise, while Longeveron’s mid-stage setback prompted questions about its future.

Not a month after reports of three deaths in autoimmune trials for Novartis’ CAR T cell therapy ignited a debate about the use of these treatments in nonfatal diseases, Kyverna Therapeutics reported data bolstering its case to bring to market the first CAR T for an autoimmune disease.

A surge of consolidation, rising appetite for specialized manufacturing and a shift toward decentralized trials is reshaping Switzerland’s life sciences landscape. From peptide producers to organoid CROs, service providers are scaling and diversifying as global sponsors turn to Swiss hubs for talent, innovation and regulatory strength.

Jefferies analysts made the prediction after Sarepta Therapeutics, which has endured nothing short of a saga with the Duchenne muscular dystrophy gene therapy Elevidys, presented data this week showing its clinically meaningful benefit in older patients who are able to walk.

Ebvallo, which Pierre Fabre Pharmaceuticals and Atara Biotherapeutics are proposing to treat a posttransplant complication, joins a wave of rejected or discouraged therapies on which the FDA has reversed previous guidance after the exit of former Commissioner Marty Makary.

Summit Therapeutics scores a $2 billion equity investment from AstraZeneca to support ivonescimab’s late-stage development; uniQure crashes after four-year data from its Huntington’s disease gene therapy failed to impress; Kyverna Therapeutics brings new light to a struggling CAR T space in autoimmune disease.

Stem cell therapies vary widely yet many physicians still treat them as interchangeable. A more credible model requires diagnostics, product characterization and conventional medical context to determine who and when should receive regenerative treatment.

Ten patients out of 20 who received Johnson & Johnson and Legend Biotech’s Carvykti remained alive at five years of follow up.