Cell therapy weekly: world’s first hospital-based autologous foundry
This week: Regeneron Pharmaceuticals (NY, USA) provided an update on the Phase I/II CHORD trial for its hearing loss gene therapy, while the first patient was dosed with Inceptor Bio’s (NC, USA) CAR-T therapy for clear cell renal cell carcinoma (ccRCC). Plus, the world’s first hospital-based autologous iPSC foundry is in the works.
The news highlights:
- Positive update on hearing loss gene therapy trial
- World’s first hospital-based autologous iPSC foundry
- First patient dosed in ccRCC CAR-T therapy trial
Positive update on hearing loss gene therapy trial
At the Research in Otolaryngology’s 48th Annual MidWinter Meeting (22–26 February 2025; Orlando, FL), Regeneron Pharmaceuticals provided an update on the Phase I/II CHORD trial for DB-OTO, an investigational gene therapy for congenital hearing loss due to OTOF gene variants.
48-week results from the first dosed individual showed “near-normal hearing across key speech frequencies” from auditory brainstem responses and speech perception tests. Out of the 11 participants with post-treatment assessments, 10 patients showed improved hearing, with three achieving near-normal or normal hearing levels by 24 weeks.
DB-OTO and its surgical procedure were well tolerated, with no serious treatment-related adverse events. Five participants experienced temporary vestibular symptoms (e.g., nausea, dizziness and vomiting), which resolved within six days.
“A year after treatment in one ear with DB-OTO, a child born profoundly deaf was able to enjoy music, engage in imaginative play and participate in bedtime reading when the cochlear implant on their other ear was removed,” elaborateed Jay Rubinstein, a CHORD clinical trial investigator. “These seemingly small interactions are life-changing for these children as well as their families and these results continue to underscore the revolutionary promise of DB-OTO as a potential treatment for otoferlin-related hearing loss.”
World’s first hospital-based autologous iPSC foundry
Cellino and Mass General Brigham’s Gene and Cell Therapy Institute (both MA, USA) are launching the world’s first hospital-based autologous iPSC foundry at Massachusetts General Hospital (USA).
Powered by Cellino’s AI-driven Nebula technology, the foundry will manufacture high-quality, patient-specific stem cells and derivative therapies on-site. Nebula’s autonomous, closed cassette system is designed to ensure precise, scalable and contamination-free production, supporting a future network of decentralized biomanufacturing hubs for personalized cell and tissue therapies.
“Centralized production of cell and gene therapies is not only expensive but also limits scalability,” explained Nathan Yozwiak, Head of Research, Mass General Brigham’s Gene and Cell Therapy Institute. “Decentralizing manufacturing through autonomous, on-site facilities may significantly cut costs and shorten the timeline from early clinical research to widespread commercial application.”
First patient dosed in ccRCC CAR-T therapy trial
Inceptor Bio has dosed the first patient in a clinical trial of IB-T101, its autologous CD70 CAR-T therapy for ccRCC. The therapy utilizes Inceptor Bio’s novel platform, OUTLAST™, to metabolically reprogram T cells to develop in the hostile tumor microenvironment through conditioning.
The clinical-stage biotechnology company also closed a US$21 million Series A2 financing led by Kineticos Life Sciences (NC, USA) to support IB-T101’s clinical development and expand its next-generation cell therapy pipeline.
“Dosing the first patient in a clinical trial is a significant milestone for any biotech company and we are proud to support Inceptor Bio as they advance IB-T101 into the clinic,” said Shailesh Maingi, Managing Partner of Kineticos Life Sciences. “The OUTLAST platform represents a compelling approach to addressing key challenges in CAR-T therapy and we believe this program has the potential to make a real difference for patients with solid tumors.”
This webinar will discuss the challenges faced in bringing iPSC-derived cell therapy from bench to patients, the solutions Catalent Pharma Solutions (NJ, USA) offers and how global partnerships can accelerate the journey from innovation to patient care.