Cell and Gene Therapy

The patient-specific nature of autologous cell therapies presents unique challenges that can best be addressed by a middle path between on-site and centralized manufacturing.

Capsida has yet to disclose the exact cause of death. The patient had received the gene therapy CAP-002 for a type of epilepsy.

Ori Biotech’s CEO said the prioritization of review by FDA, coupled to the impact of the technology, could shave up to three years off development timelines.

As AAV9 and CRISPR programs navigate safety, delivery and scalability hurdles, small molecules offer a deployable, scalable bridge, complementing genetic approaches and accelerating meaningful impact for patients with Duchenne muscular dystrophy.

It can cure deadly diseases, save long-term healthcare costs and transform lives. But the U.S. insurance system still isn’t ready to pay for it.

Vertex Pharmaceuticals commits $45 million upfront to leverage Enlaza Therapeutics’ War-Lock platform to create drug conjugates and T cell engagers for autoimmune diseases and gentler conditioning for sickle cell/beta thalassemia gene-editing therapy Casgevy.

Appia Bio’s shutdown continues an unfortunate trend this year that has seen biotech closures nearly every month.

In late May, a patient died after receiving Rocket Pharmaceuticals’ investigational gene therapy for Danon disease, spurring the hold. After lowering the dose and changing the regimen of immune modulators patients receive, the company has received FDA clearance for the trial to continue.

The FDA has postponed its decision date for Regenxbio’s Hunter syndrome gene therapy to review additional longer-term clinical data for the asset.

Kriya is advancing a host of gene therapies for a wide variety of chronic diseases, including geographic atrophy, trigeminal neuralgia and type 1 diabetes.