Around the Helix: Cell and Gene Therapy Company Updates – January 14, 2026

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.

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1. FDA Hits Atara Biotherapeutics’ T-Cell Immunotherapy Tabelecleucel for EBV+ PTLD With Second CRL

The FDA has issued a new complete response letter to Atara Biotherapeutics' regarding its biologics license application (BLA) for allogeneic Epstein-Barr virus (EBV)–specific T-cell immunotherapy tabelecleucel (tab-cel; marketed as Ebvallo) for the treatment of patients 2 years or older with EBV-positive posttransplant lymphoproliferative disease (EBV+ PTLD) who have received at least 1 prior therapy.

2. Brian Lin, PhD, on Using Genetics, Biomarkers, and Clinical Trial Readiness to Enhance ALS Care

In the lead up to the Muscular Dystrophy Association (MDA) Clinical & Scientific Conference’s upcoming meeting for 2026, Brian Lin, PhD, research portfolio director at the MDA, spoke with CGTLive’s sister site NeurologyLive^® about the ALS sessions to be presented at the upcoming conference. Throughout the discussion, Lin described a shift toward more personalized approaches in ALS, where genetic profiling, and emerging biomarkers are helping clinicians and researchers better target therapies and assess treatment responses.

3. Steven W. Pipe, MD, on Integrating Gene Therapy into Clinical Care for Hemophilia B

CGTLive spoke with Steven W. Pipe, MD, a professor of pediatric hematology/oncology at the University of Michigan Health, about practical considerations for the integration of CSL Behring/uniQure’s etranacogene dezaparvovec (marketed as Hemgenix) into real-world use.

4. Patients Treated With REGENXBIO’s DMD Gene Therapy RGX-202 Exceed Expected Disease Trajectory on NSAA

Among 4 patients treated with REGENXBIO’s RGX-202, an investigational adeno-associated virus (AAV) vector-based gene therapy intended to treat Duchenne muscular dystrophy (DMD), in the phase 1/2 portion of the AFFINITY DUCHENNE clinical trial (NCT05693142), all 4 surpassed expected disease trajectory on the North Star Ambulatory Assessment (NSAA) with utilization of the Collaborative Trajectory Analysis Project (cTAP) disease progression model at 18 months posttreatment.

5. FDA Makes its Approach to CMC Requirements More Flexible for Cell and Gene Therapy Candidates

The FDA has released a new set of “flexible” chemistry, manufacturing, and control (CMC) requirements for cell and gene therapy candidates, with the intent of pushing forward innovation and speeding up development times for these advanced therapeutics. “Regulatory flexibility must be tailored for cell and gene therapies,” FDA Commissioner Marty Makary, MD, MPH, said in a statement. “These are common-sense reforms that will address the unique characteristics of cell and gene therapies and foster more innovation.”

6. AskBio Licenses Gene Therapy Component From Genethon for Pompe Disease Program

Genethon and Bayer AG subsidiary AskBio have signed a licensing agreement through which AskBio will obtain exclusive access to use a gene therapy approach developed by Genethon in its development of a gene therapy candidate for Pompe disease. “Genethon is delighted with this agreement with AskBio, which enables the development of a gene therapy for patients suffering from this particularly severe condition,” Frédéric Revah, PhD, the CEO of Genethon, said in a statement.