New collaborations add coordinated clinical research, manufacturing, regulatory strategy and access expertise to support advancement of stalled ultra-rare disease programs

CAMBRIDGE, Mass., June 23, 2026 /PRNewswire/ — Orphan Therapeutics Accelerator (OTXL), a non-profit biotech focused on completing development and enabling commercial access to shelved rare disease treatments, today announced the expansion of its development network, Orphan ClinDevNet, through the addition of four strategic partners: OPIS, Uniphar, SK pharmteco, and TMC Pharma.

The expansion adds targeted capabilities across clinical trial execution, expanded access, manufacturing, regulatory strategy, and commercial planning, strengthening the infrastructure needed to advance promising ultra-rare disease therapies toward patients in a scalable and sustainable way.

OTXL works with rare disease, government and industry leaders to address urgent needs and obstacles that prevent promising treatments for ultra-rare diseases from advancing through approval and to patients. These therapies often stall because patient populations are too small and related risks and costs are too large to make the case for continued development via the traditional biopharma model.

Orphan ClinDevNet helps overcome these barriers by bringing together an aligned network of specialized partners that can efficiently support multiple programs across critical stages of development, lower upfront costs, de-risk the path to market, and offer alternative pathways to patient access.

“Traditional biopharma development and commercial models were created for highly competitive and crowded big disease areas, where you build and control internal R&D, manufacturing and commercial infrastructure,” said Craig Martin, CEO and Co-founder, Orphan Therapeutics Accelerator. “Rare diseases require more decentralized, efficient, out-sourced, market-adapted solutions, which is what Orphan ClinDevNet is designed to provide. By expanding this network, we are strengthening our ability to move multiple therapies forward more efficiently and bring more treatment options to highly dispersed patient populations across markets around the world.”

The addition of four new partners further expands OTXL’s growing ecosystem by adding targeted capabilities and geographic reach across areas of anticipated need for ultra-rare disease programs:

OPIS adds global clinical development and trial execution expertise across Europe, Asia, and the Americas. With extensive experience in rare disease research, strong regional operational networks, and deep regulatory knowledge, OPIS strengthens OTXL’s ability to support complex clinical programs requiring specialized study design, multinational coordination, and efficient execution across diverse regulatory environments.
Uniphar adds expanded access program capabilities to ClinDevNet. With experience in expanded access, pharmaceutical distribution, healthcare logistics, and related support services, Uniphar can help support patient access in regions where treatment pathways can be difficult to navigate. Its expertise is especially relevant for advanced therapies, where patients may need to reach specialized treatment centers and providers may require additional coordination, training, and support.
SK pharmteco adds U.S.-based manufacturing capabilities for viral vector platforms. As a global contract development and manufacturing organization, SK pharmteco strengthens ClinDevNet’s ability to support advanced therapy programs that require viral vector manufacturing expertise, including progression from development toward later-stage and commercial supply needs.
TMC Pharma adds UK and European market access, commercialization, regulatory, and consultative CRO capabilities. With a UK presence and distinct regulatory and commercialization expertise, TMC Pharma brings capabilities that are expected to align well with clinical programs OTXL is currently evaluating, particularly those requiring earlier integration of clinical strategy, regulatory planning, and market access considerations in the UK and Europe.

Partner Perspectives

Laura Ambroseli, CEO of OPIS, said: “Rare disease development is inherently complex, requiring strong alignment across scientific, regulatory, and operational functions. We are proud to contribute our global clinical development expertise to help accelerate the path from research to patients.“

Brian O’Shaughnessy, CCO of Uniphar, said: “In many markets, access is where promising therapies either reach patients or stall completely. We value being part of initiatives that focus on making that pathway more reliable so treatments can actually get to the people who need them.”

Joerg Ahlgrimm, CEO of SK pharmteco, said: “For patients with rare diseases, every second counts. That’s why we’re shortening the timeline from discovery to production by integrating manufacturing and development from day one. We are pleased to partner with OTXL to break down traditional barriers, accelerate clinical timelines, and deliver life-changing therapies to the patients who need them most.”

Simon Estcourt, CEO of TMC Pharma, said: “Development, regulation, and access are still too often disconnected from one another. We’re excited to contribute to and collaborate on efforts that bring these elements closer together earlier in the development process.”

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
[email protected]

About OPIS

OPIS is a global full-service Contract Research Organization (CRO) supporting pharmaceutical, biotechnology, and medical device companies with clinical development, regulatory, and operational services worldwide. Through extensive experience in rare and ultra-rare diseases and a network spanning Europe, Asia, and the Americas, OPIS helps sponsors execute complex multinational development programs.

About Uniphar

Uniphar is a global partner to pharma and medtech manufacturers, working to improve patient access to medicines in Europe and around the world. The company supports biopharma partners with services including expanded access program management, supply chain coordination, and healthcare logistics.

About SK pharmteco

SK pharmteco is a global contract development and manufacturing organization (CDMO) providing end-to-end services across process development, clinical and commercial manufacturing, and analytical testing. The company supports a range of advanced therapy modalities, including viral vector-based gene and cell therapies, with operations across the United States, Europe, and Asia.

About TMC Pharma

TMC Pharma is a global pharma services company partnering with small to mid-sized biotech and pharma companies to accelerate drug development timelines and maximize value across the full product lifecycle. The company provides expertise in clinical strategy, regulatory affairs, and market access, with a focus on programs in the United States, United Kingdom and European markets.

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