Manufacturing, Cell Therapies

Panelists from FDA, Genentech, and Minaris discussed raw material risk, supplier oversight, and at-risk release strategies for advanced therapies at the PDA/FDA Joint Regulatory Conference.

Four cell therapy developers have pulled back from ex vivo manufacturing in the past month — ArsenalBio, TScan Therapeutics, and Cellectis all pivoted to in vivo approaches, while Bristol Myers Squibb terminated its manufacturing partnership with automation specialist Cellares after determining its platform couldn't meet requirements for commercial Breyanzi production, triggering layoffs.

Bristol Myers Squibb reported positive topline phase 2 results for arlocabtagene autoleucel, a potential first-in-class GPRC5D-directed CAR T cell therapy, in patients with quadruple-class exposed relapsed and refractory multiple myeloma who had already received a prior BCMA-targeted therapy.

TScan Therapeutics is reorganizing to prioritize in vivo-engineered TCR-T therapy for solid tumors, pausing its phase 3 trial and autoimmune program and cutting about 75% of its workforce, even as new phase 1 data showed complete donor chimerism in all tracked heme malignancy patients.

ArsenalBio is halting all development of its ex vivo CAR T programs for solid tumors and laying off the majority of its workforce as it pivots the company entirely toward in vivo CAR T therapy, joining a fast-growing field that already includes Johnson & Johnson, Eli Lilly, and several other developers.

Celularity has entered a US manufacturing collaboration with MuseCell Innovations to produce Dezawa MuseCells and related exosome and secretome products at Celularity's Florham Park, New Jersey facility, a relationship that could generate more than $300 million in aggregate purchases over five years.

HaemaLogiX has dosed the first patient in its Phase 1 KOALA trial of KMCAR T-cell therapy, a novel CAR-T designed to selectively target kappa-restricted multiple myeloma cells while sparing healthy immune cells, with the treatment reported as well tolerated and a second patient enrolling at a higher dose.

4basebio and Genezen have entered an expanded, non-exclusive collaboration giving drug developers access to 4basebio's hpDNA cell-free synthetic DNA technology within Genezen's viral vector development and manufacturing services, aiming to streamline gene and cell therapy programs from early development through commercial manufacturing.

AbelZeta Pharma has reclaimed global rights to prizloncabtagene autoleucel from Janssen and secured FDA clearance to begin US trials, positioning the anti-CD20/CD19 bispecific CAR-T for patients who relapse after existing CAR-T therapies.

NTx Bio's NTxscribe CORE platform is being used by Baylor College of Medicine to manufacture mRNA for DOC1021, Diakonos Oncology's personalized dendritic cell vaccine, now dosing patients in a Phase 1 trial for refractory melanoma.

Outsourced cell and gene therapy manufacturing is set to grow nearly 6-fold by 2035 as biotech pipelines outpace in-house capacity.

Fate Therapeutics has initiated RECLAIM-LN, a Phase 2 potentially registrational trial of its off-the-shelf, iPSC-derived CD19-targeting CAR T-cell candidate FT819, in patients with refractory lupus nephritis, following favorable Phase 1 safety and efficacy data.

The FDA has cleared Satellite Bio's IND application for SB-101, an off-the-shelf hepatocyte cell therapy for urea cycle disorders, marking the company's transition to clinical-stage status ahead of a planned Phase 1/2 trial in infants later this year.

Kyverna Therapeutics and ElevateBio have entered a commercial manufacturing and supply agreement for miv-cel, Kyverna's autologous CD19-targeting CAR T-cell therapy, securing scalable production ahead of a planned launch in stiff person syndrome.

Johnson & Johnson has entered a collaboration and equity investment agreement with Sail Biomedicines to advance in vivo CAR-T therapies for immune-mediated diseases, securing an exclusive option to acquire the company for $2.58 billion.

Ahead of a July 29 FDA advisory committee meeting, the FDA released briefing documents disputing Capricor Therapeutics' claim that its Phase 3 HOPE-3 trial of deramiocel, a cell therapy for Duchenne muscular dystrophy cardiomyopathy, met its primary and secondary efficacy endpoints, a disagreement centered on which version of the trial's statistical analysis plan should govern the results.

MilliporeSigma's Sebastián Arana outlines why flexible, platform-based manufacturing and workforce training are now table stakes for competitive biomanufacturers.

Umoja Biopharma has received FDA clearance of its IND application for UB-VV400, a CD22-directed in vivo CAR T-cell therapy candidate built on the company's VivoVec lentiviral platform, positioning it as the industry's first known in vivo CAR T program targeting CD22 for relapsed or refractory B-cell malignancies.

Allogene Therapeutics has published complete phase 1 data in the Journal of Clinical Oncology for ALLO-316, a CD70-targeted allogeneic CAR T therapy, showing a 31% confirmed response rate and durable remissions in patients with high CD70-expressing metastatic renal cell carcinoma.

The FDA has granted Regenerative Medicine Advanced Therapy designation to Allocetra, Enlivex's macrophage-reprogramming cell therapy, for age-related symptomatic knee osteoarthritis in patients 64 and older, based on Phase I/IIa data showing durable pain and function improvements.

FT839, Fate Therapeutics' off-the-shelf, iPSC-derived CAR T-cell candidate co-targeting CD19 and CD38, has received FDA IND clearance and will advance into a phase 1/2 basket trial designed to treat multiple autoimmune diseases without conditioning chemotherapy.

Cytiva’s Pierre-Alain Ruffieux discusses standardization, regionalization, talent gaps, and regulatory pathways shaping advanced biomanufacturing’s future ahead of BIO 2026.

Made Scientific and Pluristyx will integrate iPSC starting materials with CDMO services for cell therapy development.

Charles River Laboratories’ Dr. Alan K. Smith explains how early commercial planning, modular manufacturing, and AI-driven automation may accelerate scalable manufacturing for cell and gene therapies.

Cell therapy developers are prioritizing scalable manufacturing strategies alongside efficacy to support broader commercial deployment, notes Cellular Origins CEO Dr. Edwin Stone at the 2026 ASGCT Annual Meeting.