GeneFab, a contract research, development, and manufacturing organization (CRDMO) specializing in cell and gene therapies, has formed a strategic partnership with RegCell to advance innovative regulatory T cell (Treg) therapies for autoimmune diseases into US clinical trials. The collaboration will facilitate the technology transfer of RegCell's proprietary Treg platform from Japan to GeneFab's state-of-the-art manufacturing facility in Alameda, California.
Under the agreement, GeneFab will provide comprehensive CRDMO support, including technology transfer, process development, scale-up, IND-enabling activities, and clinical-grade manufacturing. The partnership aims to accelerate RegCell's path to US clinical trials, with an initial focus on autoimmune hepatitis.
Strategic Manufacturing Partnership
GeneFab's 92,000 square foot Alameda facility, equipped with seven GMP clean rooms, will serve as the manufacturing hub for RegCell's Treg therapies. The facility's location in the San Francisco Bay Area provides proximity to RegCell's leadership team, facilitating close collaboration throughout the development process.
"This collaboration embodies what we do best, partnering with cutting-edge therapeutic developers to accelerate their journey to the clinic," said Dr. Philip Lee, CEO at GeneFab. "RegCell has developed a best-in-class platform to generate stable, epigenetically reprogrammed, antigen-specific Tregs, and our team is proud to help bring that momentum to the US."
The partnership extends beyond manufacturing support, with both companies exploring co-development opportunities to optimize Treg therapies using GeneFab's expertise in synthetic biology. "These initiatives may include integrative designs and optimizations aimed at improving the safety and potency of the next generation of therapies," explained Dr. Duncan Liew, Senior VP of Business Development at GeneFab.
RegCell's Novel Approach to Autoimmune Diseases
RegCell's technology focuses on epigenetic reprogramming of regulatory T cells, which play a critical role in maintaining immune tolerance. Unlike conventional treatments for autoimmune diseases that rely on broad immunosuppression, RegCell's approach aims to restore immune tolerance through antigen-specific Tregs.
The company was founded based on the pioneering discoveries of Professor Shimon Sakaguchi, a leader in regulatory T-cell biology. RegCell's platform addresses key challenges that have hindered the development of autoimmune cell therapies, particularly the issues of unknown or multiple pathogenic antigens.
"This partnership marks the next step of our mission to bring our groundbreaking immunology therapy to market and transform outcomes for patients with autoimmune diseases," said Dr. Michael McCullar, CEO of RegCell. "Working with GeneFab feels like an extension to our team. Its end-to-end capabilities, coupled with its specialist expertise in the design, manufacture, and commercialization of cell therapies, enables us to develop the foundation of a commercially viable manufacturing process."
Significant Funding and US Transition
The partnership announcement follows RegCell's recent financial and operational developments. The company has secured $45.8 million in funding, including $8.5 million from an oversubscribed seed round co-led by The University of Tokyo Edge Capital Partners (UTEC) and Fast Track Initiative (FTI), with participation from several other investors.
Additionally, RegCell received up to $37.3 million in non-dilutive funding from Japan's Agency for Medical Research and Development (AMED), underscoring Japan's commitment to breakthrough therapies. This substantial financial backing will support the development of RegCell's platform through clinical proof-of-concept.
The company has also completed its transition to a US-based entity, establishing headquarters in Emeryville, California. This strategic move aims to accelerate clinical development, foster global partnerships, and enhance commercialization opportunities while maintaining core scientific operations in Japan.
Clinical Development Timeline
RegCell plans to initiate first-in-human clinical trials in 2025, targeting an antigen-specific autoimmune indication. The AMED funding will support IND-enabling studies, GMP optimization, and establishing clinical proof-of-concept in the US.
"This funding accelerates RegCell's mission of developing potentially curative treatments for autoimmune diseases and transplantation and provides a very efficient path to clinical proof of concept," stated Professor Sakaguchi.
GeneFab's Expansion Plans
As part of its growth strategy, GeneFab is investing in automation platforms and scaling capabilities to support pivotal trials and commercial readiness. The company plans to build out an additional 45,000 square feet at its Alameda site, aligning with its vision of enabling cost-effective and scalable access to advanced cell therapies.
This expansion will strengthen GeneFab's position as a local manufacturing hub for clinical-stage biotech and early-stage innovator companies in the Bay Area and beyond, particularly those developing next-generation cell and gene therapies.
The collaboration between GeneFab and RegCell represents a significant step forward in bringing innovative Treg-based therapies to patients with autoimmune diseases, potentially offering a more targeted and effective alternative to current treatment options.
