AGC Biologics Joins Orphan Therapeutics Accelerator’s Clinical Development Network as a Manufacturing Partner to Advance Cell and Gene Therapies for Ultra-Rare Diseases

Collaboration brings AGC Biologics’ advanced therapy expertise and global footprint to OTXL’s network, supporting a sustainable path from clinical development to commercial access for therapies serving the smallest patient populations

CAMBRIDGE, Mass., May 11, 2026 (GLOBE NEWSWIRE) -- Orphan Therapeutics Accelerator (OTXL), a non-profit biotech focused on completing development and enabling commercial access to stalled rare disease treatments, today announced that AGC Biologics, a leading global biopharmaceutical Contract Development and Manufacturing Organization (CDMO), has joined OTXL’s Clinical Development Network (Orphan ClinDevNet) as a manufacturing partner. The strategic alliance expands existing collaboration between the two organizations and strengthens OTXL’s ability to advance cell and gene therapies for ultra-rare conditions with coordinated, end-to-end manufacturing capabilities.

OTXL’s Orphan ClinDevNet brings together specialized partners committed to overcoming the most persistent barriers to development and commercialization of ultra-rare disease treatments, from the cost and pace of late-stage clinical trials to the difficulty of maintaining continuity and scaling manufacturing once a therapy is approved. OTXL and its network partners work together to help lower upfront development costs, de-risk the path to market, and enable more therapies to reach patients.

“Despite major advances in cell and gene therapy, the commercial sustainability of treatments for ultra-rare diseases remains a critical hurdle, preventing many proven therapies from reaching patients,” said Luca Alberici, Vice President of the Cell and Gene Technologies Division at AGC Biologics. “At AGC Biologics, we have built the specific expertise and infrastructure to sustainably manufacture these products, demonstrated by our success with numerous commercial CD34+ HSC-based therapies. Our alliance with OTXL expands our network of partners dedicated to bringing these life-changing treatments to patients by creating a new, viable model for the rare disease ecosystem.”

“We’ve gotten to know AGC Biologics as the manufacturing partner to Fondazione Telethon for WASKYRA™, as we work together toward the US commercial launch,” said Craig Martin, CEO and Co-founder, Orphan Therapeutics Accelerator. “We are pleased to now have the opportunity to expand that relationship through Orphan ClinDevNet. AGC’s expertise in ex vivo gene therapies and its ability to scale production through commercial supply gives us a manufacturing foundation that can support not only WASKYRA but also additional shelved programs we take on.”

The team at AGC Biologics’ Milan site brings more than three decades of experience in complex biologics manufacturing and a track record of 11 viral vector and 7 cell therapy commercial approvals from the European Medicines Agency, the UK Medicines and Healthcare products Regulatory Agency, and the U.S. Food and Drug Administration. Through its longstanding work with Fondazione Telethon, AGC Biologics has developed and produced clinical-grade lentiviral vectors and patient-specific genetically engineered cells from preclinical through commercial manufacturing, including for WASKYRA.

When WASKYRA was approved in December 2025, Fondazione Telethon became the first non-profit organization to receive FDA approval of a gene therapy, with AGC Biologics as its chosen manufacturer and Orphan Therapeutics Accelerator’s subsidiary Orphan Therapies as its US commercialization partner.

AGC Biologics’ cell and gene therapy platform, anchored in autologous CD34+ hematopoietic stem and progenitor cells transduced with lentiviral vectors, is directly applicable to the kinds of programs OTXL is onboarding. The collaboration also builds on AGC Biologics’ prior work supporting therapies that had been considered commercially nonviable under traditional industry practices due to small patient populations and high per-patient manufacturing costs.

“AGC Biologics’ global footprint extends our ability to connect with patients and treatment centers across more geographies. This is a major benefit when serving populations as small and dispersed as those affected by ultra-rare diseases, as ultimately, we want to get therapies to the patients who need them, wherever they may be,” added Martin.

About Orphan Therapeutics Accelerator (OTXL)

The Orphan Therapeutics Accelerator (OTXL) is a patient-centered non-profit biotech focused on obtaining and reinitiating development of promising “shelved” clinical-stage treatments for ultra-rare conditions and providing a scalable and sustainable commercialization path to get these treatments to patients. OTXL leverages its non-profit status, success-based agreements and incentives to obtain rights to shelved programs and complete development at deferred or lower cost via a network of affiliated CDMOs, CROs and other partners. When a program is approved and generates revenue, net proceeds are used to repay and reward contributing partners, companies, and institutions, with a portion returned to the Orphan Therapeutics Accelerator to fund additional programs. Our team is deeply familiar with challenges faced in developing and commercializing treatments for rare conditions and committed to overcoming them. For more information, please visit www.orphantxl.com.

Media Contact
FINN Partners for Orphan Therapeutics Accelerator
Nicole Grubner
nicole.grubner@finnpartners.com
929 588 2011

About AGC Biologics

AGC Biologics is a leading global biopharmaceutical Contract Development and Manufacturing Organization (CDMO) with a strong commitment to delivering the highest standard of service as we work side-by-side with our clients and partners, to provide friendly and expert services. We provide world-class development and manufacturing of mammalian and microbial-based therapeutic proteins, plasmid DNA (pDNA), messenger RNA (mRNA), viral vectors, and genetically engineered cells. Our global network spans the U.S., Europe, and Asia, with locations in Seattle, Washington; Copenhagen, Denmark; Heidelberg, Germany; Milan, Italy; and Chiba and Yokohama, Japan. AGC Biologics is a part of AGC Inc.’s Life Science Business. The Life Science Business runs 10+ facilities focused on biopharmaceuticals, advanced therapies, small molecule active pharmaceutical ingredients, and agrochemicals. To learn more, visit www.agcbio.com.

Media Contact

AGC Biologics
Kati Sills
Senior Manager, Public Relations
kati.sills@agc.com

AGC Inc. Corporate contact:
info-pr@agc.com

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