Unlocking Stranded Innovation for Children: How CobiCure Is Reimagining Access to Life-Changing Therapies

Across the biomedical landscape, promising therapies for rare and ultra-rare diseases are being abandoned, not because they fail scientifically, but because they fail commercially. This is especially devastating for pediatric patients who often face progressive, life-limiting conditions with no alternatives. When programs stall due to the economics of small patient populations, innovation becomes stranded and children pay the price.

We recently met with David Barrett, Chief Executive Officer of ASGCT (American Society of Gene & Cell Therapy), and Terence Flotte, President, to discuss what the organization is doing to advance stalled innovation and learned that there are many areas of shared interest. They recently published a white paper that reframes these therapies as “commercially pre-viable” rather than “non-viable,” emphasizing that with the right infrastructure, including regulatory flexibility, manufacturing efficiencies, and alternative financial models, these programs can and should reach patients. Their proposed solutions, including an IP repository for discontinued programs and a consortium to achieve economies of scale, represent critical steps toward systemic change.

At CobiCure Therapeutics, our mission is to ensure that children with cancer and rare genetic diseases are not left behind. We share ASGCT’s conviction that value must be defined by clinical impact, not market size. To that end, we are building a model specifically designed to rescue stranded innovation and accelerate therapies for pediatric patients who have no time to wait. We aim to achieve this by:

• Seeking stranded innovation: Like ASGCT’s proposed repository, we are working to identify and acquire promising assets that have been deprioritized for commercial reasons, ensuring they do not disappear into IP limbo.
• Assembling expert teams on demand: Rather than maintaining a large internal infrastructure, we curate external experts and capabilities on a per-project basis. This approach minimizes fixed costs while ensuring access to world-class scientific, regulatory, and manufacturing expertise.
• Leveraging proven drug development practices: Founded within and adjacent to a robust healthcare investment ecosystem, we apply best practices that have been road-tested for advancing drugs to the clinic in the for-profit world without having to face the steep learning curve typical of new ventures.
• Convening a syndicate of aligned funders: We bring together philanthropic organizations committed to meeting the needs of underserved pediatric populations and impact investors who value addressing unmet needs with modest but meaningful returns. This blended capital model enables sustainability without relying on blockbuster economics.

The ASGCT framework validates the urgency and feasibility of nontraditional models. It also highlights the need for trusted intermediaries, entities that can broker IP transfers, convene stakeholders, and maintain development continuity. CobiCure is positioned to play that role, not as a passive repository, but as an active developer and accelerator of rare disease programs focused on pediatric patients.

The question posed by ASGCT, “How the global ‘we’ choose to face this moment will define the next 50 years of innovation”, is the question we ask ourselves every day. For children with cancer and rare genetic diseases, the cost of inaction is measured in lives and lost potential. By operationalizing the principles outlined in this white paper, CobiCure is building a model that ensures scientific breakthroughs translate into accessible treatments for the youngest and most vulnerable patients.

Read the ASGCT white paper here and learn more about how CobiCure is redefining what is possible for pediatric rare disease therapies.