Around the Helix: Cell and Gene Therapy Company Updates – December 3, 2025

The cell and gene therapy sectors are growing exponentially, with new players emerging daily and much progress being made both in and out of the lab. CGTLive®’s Around the Helix is your chance to catch up with the latest news in cell and gene therapies, including partnerships, pipeline updates, and more.

Have a cell and gene therapy news update you’d like to share with our editorial team? Tag us on social (@CGT_Live on X, or CGTLive on LinkedIn) and use #AroundTheHelix!

1. Structuring Partnerships Among Academia, Biotech, and Pharma

Deborah Phippard, PhD, the chief scientific officer of Precision for Medicine, and Renier Brentjens, MD, PhD, the chair of the department of medicine and the deputy director at Roswell Park Comprehensive Cancer Center, shared their thoughts on how the different entities that contribute to cell and gene therapy research and development can better collaborate.

2. ImmunoLogic, Episode 7: "Turbocharging CAR T-Cells: The IL-9 Orthogonal Signal” With Anusha Kalbasi, MD

In this episode, the cohosts speak with Anusha Kalbasi, MD, an associate professor of radiation oncology (radiation therapy) at Stanford Medicine, about his recently published paper on the use of interleukin-9 (IL-9) to enhance chimeric antigen receptor T-cell (CAR-T) therapy efficacy without the need for lymphodepleting chemotherapy.

3. FDA Activity Recap: November 2025 Features SMA Approval, Boxed Warning for Elevidys, and More

Our team highlighted several important FDA actions from last month, such as the approval of Novartis’ intrathecally-delivered spinal muscular atrophy gene therapy onasemnogene abeparvovec-brve and the addition of a boxed warning to the prescribing information for Sarepta Therapeutics' Duchenne muscular dystrophy (DMD) gene therapy delandistrogene moxeparvovec-rokl (marketed as Elevidys).

4. Karlla W. Brigatti, MS, CGC, on Accelerating SMA Diagnosis and Treatment

In an interview with CGTLive®, Karlla W. Brigatti, MS, CGC, the research director at the Clinic for Special Children, outlined the Clinic’s efforts to improve outcomes for infants with spinal muscular atrophy (SMA) in the Amish and Mennonite communities.

5. Capricor's Pivotal Phase Trial for DMD Cell Therapy Hits Primary End Point

Capricor Therapeutics' phase 3 HOPE-3 clinical trial (NCT05126758), which is evaluating investigational allogeneic cardiosphere-derived cell therapy deramiocel for the treatment of DMD, has hit its primary end point, with a 54% slowing of progression seen for deramiocel versus a placebo on the Performance of Upper Limb Total Score (n=105, P = .029). The percent slowing was calculated as the treatment difference divided by the change from baseline for the placebo.

6. Latus Bio's CLN2 Disease Gene Therapy Cleared for US Trial

Latus Bio has received clearance of an investigational new drug (IND) application from the FDA for LTS-101, an adeno-associated virus (AAV) vector-based gene therapy candidate for the treatment of central nervous system manifestations of late-infantile neuronal ceroid lipofuscinosis type 2 (CLN2) disease. Alongside the IND clearance, LTS-101 also received a slew of designations from the FDA, including orphan drug designation, rare pediatric disease designation, and fast track designation.