Cell and Gene Therapy

In this webinar, we will show how delayed isolation and cryopreservation of immune cells from fresh leukopaks can drastically impact their stability and functionality. We will also explore how purified and cryopreserved cell populations such as PBMCs and isolated T cells streamline logistics, reduce variability, and enhance transduction efficiency in CAR-T production.

Less than two weeks away from a highly anticipated FDA ruling on its cell therapy for Duchenne muscular dystrophy, Capricor Therapeutics has reported increased investment in its San Diego plant to support commercial production.

Novartis confirms that nine patients treated with its spinal muscular atrophy gene therapy Zolgensma have developed tumors; the FDA orders GLP-1 drugmakers to add a warning to their labels about the risk of a rare eye condition that can lead to vision loss; and the agency is set to decide on no fewer than 11 therapies this month, including closely watched applications from Summit Therapeutics, Vertex and Capricor Therapeutics.

Last month, the FDA committed to designing a “fit-for-purpose” framework for regulating rare disease therapies based on patient need and realistically attainable evidence. With the upcoming decision for Capricor’s Duchenne muscular dystrophy cell therapy deramiocel, this paradigm faces its first key test.

Beam Therapeutics’ risto-cel provided at least 12 consecutive months of freedom from severe pain crises for patients with sickle cell disease in a mid-stage trial, setting the stage for an FDA application as early as this year.

After Novartis confirmed the reports of tumors in patients who have been treated with the spinal muscular atrophy gene therapy, the FDA will determine if any regulatory action is needed for Zolgensma or for the pharma’s similar product Itvisma.

SK pharmteco’s Tatiana Nanda, chief technical officer of SK pharmteco Viral Vector North America discusses what gene therapy sponsors must account for sooner rather than later when preparing for GMP manufacturing.

The BioSpace 40 Under 40 honoree helped steer Forge’s sale to Ajinomoto. Now, Samantha Piteri-Molina sees opportunities in AI-driven analytics and Ohio’s gene therapy ecosystem—with a responsibility to patients worldwide.

Sarepta Therapeutics’ newly minted CEO Michael Severino laid out his path to returning the muscular dystrophy therapy maker to the forefront of the rapidly expanding market.

The majority of the large pharmas have now reported third quarter earnings—spoiler alert: Eli Lilly made more money than anyone expected. Plus, the FDA dropped another initiative aimed at accelerating rare disease treatments; HHS greenlights the compounding of six unapproved peptides; and uniQure’s embattled gene therapy for Huntington’s disease has finally been accepted for review.