Cell therapy weekly: investigational AAV-based gene therapy for FSHD completes enrollment
This week: The US Food and Drug Administration (FDA; MD, USA) cleared an Investigational New Drug (IND) application for a novel adoptive cell therapy and a first-in-human trial assessing an investigational adeno-associated virus (AAV)-based epigenetic gene therapy, indicated for facioscapulohumeral muscular dystrophy (FSHD). This completes enrollment and dosing in its dose-escalation stage. Plus, two biotechnology companies have entered into a partnership to advance scalable induced pluripotent stem cell (iPSC)-based bioprocessing solutions.
The news highlights:
- IND cleared for novel adoptive cell therapy for solid cancer
- Investigational AAV-based gene therapy for FSHD completes enrollment
- Partnerships to advance scalable iPSC-based bioprocess solutions
IND cleared for novel adoptive cell therapy for solid cancer
The FDA has cleared an IND application for CoRegen, Inc.’s (TX, USA) CRG-150 – a novel, adoptive cell therapy indicated for multiple solid tumor types. CRG-150 targets and disrupts steroid receptor coactivator 3 in regulatory T cells, which is a key gene that is often overexpressed in most human cancers. This novel approach aims to restore the immune system’s ability to recognize and eliminate solid tumors.
In preclinical studies, this approach completely eliminated tumors in multiple mouse models of difficult-to-treat solid cancers and provided long-term protection against recurrence, without causing systemic autoimmune side effects.
CoRegen is now positioned to initiate Phase I/IIa studies to assess the safety, tolerability and optimal dosing of the therapy, enrolling patients diagnosed with metastatic triple-negative breast cancer, metastatic HR+ HER2– breast cancer and metastatic prostate cancer.
“Moving into the clinic is a pivotal step, and we are doing so alongside globally renowned major academic cancer centers that will serve as sites for our Phase I study. Grounded in the vast volumes of preclinical work our team has conducted at Baylor College of Medicine, we are optimistic about what lies ahead,” relayed Chief Medical Officer, Sonal Gupta (CoRegen, Inc.). “While CRG-150 will initially be evaluated in patients with three selected tumor types, we believe the potential of this product and our broader pipeline could address critical needs across numerous cancers.”
Investigational AAV-based gene therapy for FSHD completes enrollment
Clinical-stage company Epicrispr Biotechnologies (CA, USA) announced the completion of enrollment and dosing in the dose-escalation portion of its first-in-human clinical trial assessing EPI-321 for FSHD. Twelve patients have been enrolled across two intravenous dose levels, with six patients in each cohort.
EPI-321 is an investigational, one-time AAV-based epigenetic gene therapy that has been designed to address inappropriate expression of the DUX4 gene, which leads to progressive skeletal muscle degeneration, weakness and disability. Previously reported interim clinical data demonstrated a favorable safety profile and early evidence of disease modification, with statistically significant increases in whole-body lean muscle volume and favorable outcomes in strength and function.
“Completing enrollment and dose escalation marks an important milestone for the EPI-321 program,” said Amber Salzman, CEO of Epicrispr Biotechnologies. “We completed enrollment as rapidly as the study protocol allowed in this complex first-in-human AAV gene therapy trial, and our recently reported interim data provide encouraging early evidence that EPI-321 may modify the underlying biology of FSHD. We look forward to presenting additional clinical data at the World Muscle Society Annual Congress in September and completing 12-month follow-up across all participants next year.”
The World Muscle Society Annual Congress will be held in Hiroshima, Japan from 29 September–3 October 2026.
Partnerships to advance scalable iPSC-based bioprocess solutions
Combining Good Manufacturing Process (GMP) compatible iPSC technology and engineered media systems and manufacturing expertise, RoosterBio (MD, USA) and Applied StemCell, Inc. (CA, USA) have entered into a strategic partnership to develop and commercialize scalable iPSC-based bioprocess solutions. The collaboration intends to deliver “end-to-end bioprocessing solutions that advanced therapies need to scale and reach more patients,” according to Dolores Baksh, CEO of Applied StemCell.
The solution will aim to:
- Deliver a comprehensive system with bioprocess media
- Provide iPSCs sourced from GMP-compliant starting material
- Enable manufacturing processes compatible with the scale required for clinical and commercial production
- Close consistency and quality gaps in iPSC development
- Provide integrated solutions designed to shorten process development cycles
- Support transitions from early research to scalable clinical manufacturing
- Address the global demand for scalable, high-quality iPSC-based products
“By combining Applied StemCell’s GMP-enabled iPSC lines with RoosterBio’s media, reagent and scalable bioprocess development solutions, we will deliver an end-to-end technology platform that reduces complexity, accelerates timelines, and creates transformational value for the advanced therapy industry,” reiterated RoosterBio’s CEO, Tim Kelly.
