Cell and Gene Therapy

J&J will hand over the rights to bota-vec for $25 million upfront, clearing MeiraGTx to seek regulatory approvals in the U.S. and EU in 2027.

In this whitepaper, BioSpace reviews the major trends impacting the CDMO sector and the evolving relationship between sponsors and providers. We examine the key qualities pharma and biotech should consider in CDMO selection, and how the macroeconomic and macrodevelopment factors affecting the space play a role in this selection.

The draft guidance supports the agency’s new pathway designed to speed up the development of custom gene therapies.

In this episode of Denatured, you’ll hear from Jason Jones, head of global business development at Cellular Origins and Alexander Seyf, founder & CEO of Autolomous. They discuss how the push to scale cell and gene therapy manufacturing is accelerating interest in automation, digitization, robotics and deeper collaboration across the ecosystem.

Gilead Sciences has inked three deals this year so far totaling $14.77 billion, a marked escalation of the company’s usual M&A pace. Executives detailed the rationale for buying Arcellx, Ouro Medicine and Tubulis GmbH and whether they are interested in further deals.

As cell and gene therapy developers face rising pressure to produce therapies faster and at lower cost, the industry is leaning on robotics, digital systems and partnerships to bridge the gap between innovation and delivery.

While the accelerated approval unlocks only a small market opportunity for Rocket Pharmaceuticals, it will give the biotech a chance to prepare for future product launches, according to Jefferies.

Biomarker data for Sarepta Therapeutics’ RNA programs, licensed from Arrowhead Pharmaceuticals, are “competitive” and “strong,” according to analysts at Jefferies, which projected over $1 billion in peak sales.

Following last month’s $7.8 billion purchase of CAR T biotech Arcellx, Gilead’s dealmaking train chugs along with yet another acquisition—this time securing Ouro Medicines’ pipeline of T cell engagers for inflammatory diseases.

Oryon Cell Therapies’ lead cell therapy is an autologous treatment designed to replace dopaminergic neurons in patients with Parkinson’s disease. Phase 1b/2a data showed that the asset can improve motor function and mobility in patients.