In Vivo Gene Therapy Development Program

Unleashing the potential of high-risk, high-reward innovations towards scalable treatments for blood diseases

NHLBI's Catalyze In Vivo Gene Therapy Development Program Expression of Interest submissions will begin on September 3, 2026!

Despite major advances in gene therapy and the availability of transformative treatments for sickle cell disease (SCD), millions of people worldwide living with SCD and other blood disorders still do not have access to these therapies.

To help close this gap, NHLBI has launched its In Vivo Gene Therapy Development Program, inviting the scientific community to accelerate transformative in vivo hematopoietic stem cell (HSC) and gene therapies, starting with SCD and expanding to other blood disorders.

If your concept challenges prevailing assumptions, bridges disciplines, or offers a novel approach to overcoming fundamental barriers, we want to hear from you!

Transformative gene therapies will not emerge from incremental innovation alone. We will support bold, early-stage ideas (Technology Readiness Level 1-2) that may be too high-risk for conventional funding. While not all approaches will succeed, we expect some will show enough promise to advance toward clinical trials with NHLBI’s support.

All applicants must submit an expression of interest form (EOI). Submitted EOIs are reviewed in the order that they are received. Applicants will be notified by email whether they have been approved to proceed to the next step. Applicants moving forward to full application will have 4 weeks to submit.

Promoting discovery to expand treatment options for sickle cell disease & other blood disorders

Funding

This program will initially fund a wide range of high-risk, high-reward proof of concept projects (12-18 months) that propose innovative solutions to key barriers to in vivo gene therapy targeting HSCs (with emphasis in SCD).

Focus Areas

Initial funding will target innovations focused on:

  • Novel vector platforms
  • Non-viral delivery systems
  • Immune evasion technologies
  • Translational models or assays

How do I apply?

Step 1

All applicants must submit an expression of interest form (EOI).

Online submissions will begin on September 3, 2026 and end on September 20, 2026.

You may download a preview of the EOI form:
Download EOI form (PDF) Download EOI form (Word)

Note: An EOI submission is only needed for preclinical, regulatory, and in vivo gene therapy services.

Step 2

After EOI submissions, a select group of applicants will be invited to participate in a required 1-day event at NHLBI on November 5th, 2026 to present their concept.

Step 3

Prioritized concepts will advance to a full application submission and external review.

Submit your email address so that we can contact you with Catalyze In Vivo Gene Therapy Development Program updates:

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