Craig Martin | Founder, CEO
Orphan Therapeutics Accelerator

Craig Martin, Founder, CEO, Orphan Therapeutics Accelerator

Craig is a deeply experienced executive, advisor and board director, with extensive background in biotech and health technology fields. His focus in emerging innovation and desire to make an impact have led to a longstanding commitment to supporting rare disease communities. Craig has led for-profit and non-profit organizations focused on advancing and expanding access to treatments for rare, genetic conditions and is a frequent speaker and commentator on overcoming challenges facing innovators and caregivers and patients with rare diseases. He has worked extensively on the advancement of new technologies and treatment modalities – including gene editing and gene therapy, mRNA and microbiome-based treatments, genomic sequencing and molecular diagnostics, as well as in wearables and other aspects of digital health. In June 2024, Craig founded the Orphan Therapeutics Accelerator (OTXL), a non-profit biotech focused on reinitiating development and commercializing promising treatments for ultra-rare diseases that companies and institutions have shelved due to shifting priorities, financial pressures and/or policy and other influences. In December 2025, OTXL announced a groundbreaking non-profit partnership with Fondazione Telethon to commercialize a gene therapy in the U.S. In January 2026, OTXL formed a partnership with the American Society for Gene & Cell Therapy (ASGCT) to establish CGTxchange, an AI-enabled marketplace to match shelved gene and cell therapies with prospective investors and partners to expand access to treatments for ultra-rare conditions. 

Appearances:



Day 1 - World Orphan Drug Congress USA 2026 @ 12:10

Rewriting the Playbook: Innovative Commercial Pathways for Ultra-Rare Disease Treatments

With high unmet need and extremely small patient populations, traditional commercialization and development playbooks fall short for ultra-rare diseases. This session will review several case studies in innovation, each with broad relevance to ultra-rare therapy developers. 

Objectives: 

  • The model behind the first ever non-profit commercialization of a gene therapy 
  • How digital marketplaces can accelerate the path to marketing approval and commercial partnerships 
  • The catalytic role of clinical development networks in enabling late-stage development and commercial-stage success. 
last published: 11/Mar/26 19:05 GMT

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