Nonprofit Accelerator Commits $1.7 Million to Five Projects Targeting Blindness, Sepsis and Rare Disease

A quiet but consequential shift is underway in how early-stage academic drug discovery reaches the clinic. Critical Path Institute, the Tucson-based nonprofit known for building regulatory science consortia, has announced that its Translational Therapeutics Accelerator has distributed $1,699,500 across five Bridging Research and Innovation in Drug Development Grants, known as BRIDGe awards. The grants, announced individually between March and July 2026, support projects spanning sepsis-related cognitive decline, inherited blindness, neurological lysosomal storage disease, pediatric cryptosporidiosis and neuropathic pain. All five fall within the accelerator’s three priority areas of brain health, pediatrics, and rare and orphan diseases, and together they represent every therapeutic modality the program funds: three small molecule programs, one protein-based therapeutic and one in vivo gene therapy.

The largest single award, $501,000, goes to Wesley Van Voorhis, M.D., Ph.D., at the University of Washington School of Medicine, to advance a compound called BKI-1708 toward an Investigational New Drug application for cryptosporidiosis. The candidate is a selective inhibitor of CpCDPK1, a kinase essential to the Cryptosporidium parasite. C-Path reports that the diarrheal disease causes an estimated 200,000 deaths each year, concentrated among children under five in low- and middle-income countries, and that the single marketed therapy has limited efficacy in malnourished children, the very population that bears the greatest burden. Moving a neglected-disease candidate from lead optimization to an IND package is exactly the kind of translational gap the BRIDGe program was designed to close.

At the University of Michigan, James Shayman, M.D., received $456,000 to advance BPN-25271, a brain-penetrant glucosylceramide synthase inhibitor developed with medicinal chemist Scott Larsen, Ph.D. The technical significance here rests on Shayman’s track record: he previously invented eliglustat tartrate, the only oral therapy approved globally for Gaucher disease type 1. But eliglustat does not cross the blood-brain barrier, which means it cannot address the neurological progression seen in Gaucher disease type 3, GM1 gangliosidosis, Tay-Sachs disease and Sandhoff disease. A substrate reduction therapy that reaches the central nervous system could, for the first time, offer a pharmacological option for the neuronopathic forms of these lysosomal storage disorders.

Inherited blindness is the focus of a $249,719 award to Odylia Therapeutics, a nonprofit biotechnology organization in Atlanta led by Ashley Winslow, Ph.D. The team is developing an adeno-associated virus gene therapy that delivers a functional copy of the USH1C gene to retinal cells. The clinical context is stark: children with Usher Syndrome Type 1C can receive cochlear implants to manage their hearing loss, but nothing currently prevents or reverses the retinal degeneration that follows in adolescence. AAV-mediated gene replacement has already proven itself in other inherited retinal dystrophies, and extending that strategy to USH1C-related disease could preserve sight during the years when vision loss most reshapes a young person’s independence.

Sepsis, one of the most common and least understood pathways to acquired brain injury, anchors a $250,000 award to Teresa Sanchez, Ph.D., at Weill Cornell Medicine. Her project targets a vascular mechanism involved in maintaining the blood-brain barrier with an antibody-based therapy. Up to half of sepsis survivors experience lasting problems with memory, thinking and concentration, a condition associated with a two- to fourfold increase in dementia risk and, critically, no effective treatment. If stabilizing the barrier’s endothelial integrity can blunt this cognitive aftermath, the work would open an entirely new therapeutic category for post-sepsis syndrome, a condition that hospital medicine has largely managed by observation rather than intervention.

The fifth award, $242,781, goes to Corey Hopkins, Ph.D., at the University of Nebraska Medical Center, working with Jerri Rook, Ph.D., of the Vanderbilt Center for Neuroscience Discovery. The team is developing sigma-1 receptor antagonists as nonopioid treatments for neuropathic pain and chemotherapy-induced peripheral neuropathy. Both conditions represent enormous unmet needs: neuropathic pain affects millions and is often managed inadequately with opioids or drugs originally designed for depression and epilepsy, while chemotherapy-induced peripheral neuropathy has no approved preventive or disease-modifying therapy at all. The grant specifically funds the construction of a pharmacokinetic/pharmacodynamic model to guide compound optimization, a quantitative scaffold that links drug exposure to biological effect and helps prioritize which analogues advance.

What distinguishes the BRIDGe program from a conventional seed grant is its architecture. The awards are structured around clear milestones and go/no-go decision points, aligning funding directly with each project’s specific work plan. Awardees retain complete ownership of their intellectual property while gaining a dedicated support system: each team meets monthly with experts from the accelerator and the broader C-Path organization, alongside formal evaluations from a standing Scientific Advisory Committee at the beginning, middle and end of the project. C-Path also offers flexible financial execution, helping investigators identify contract research organizations or consultants when a study requires external capabilities, and allowing a substantial portion of the grant to fund those partners directly.

The regulatory dimension may be the program’s most valuable currency. “Twenty years of consortium work has given C-Path a deep understanding of regulatory evidentiary standards and how those expectations evolve throughout the development process,” said C-Path CEO Klaus Romero, M.D., M.S., FCP. He noted that an academic team working toward its first IND package may not yet have the benefit of that regulatory experience, and studies designed without it can sometimes require additional work or repetition. By sharing this regulatory blueprint, the organization aims to help innovators anticipate what will be needed and generate the right evidence at the right time, an approach that can save years and millions of dollars in a development landscape where most academic candidates never reach an IND filing.

Maaike Everts, Ph.D., Executive Director of the Translational Therapeutics Accelerator, framed the grants as inflection points rather than endpoints. “Every promising result we see in the lab is just the first page of a much larger story,” she said. For these five teams, she explained, the science has reached a critical point where the right resources and expertise can help determine what comes next, and BRIDGe grants provide that momentum by combining essential funding with regulatory and development expertise to turn scientific potential into actionable evidence and move promising innovations closer to the patients who need them.

The accelerator funds faculty at universities and nonprofit institutions anywhere in the world, across small molecules, protein-based therapeutics and in vivo gene-based therapies, with award sizes that scale with project maturity from early lead optimization through the studies that support an IND application. The 2026 BRIDGe cycle added a dedicated funding track for polycystic kidney disease in partnership with the PKD Foundation, the first collaboration of its kind for the program, building on the Foundation’s long-standing support of C-Path’s Polycystic Kidney Disease Outcomes Consortium. Project CASK is supporting the 2026 effort with additional funding for projects in CASK-related disorders. The cycle closed to full proposals on August 12, 2026, with additional awards to be announced following review, meaning the five projects described here may be only the first page of this year’s larger story.

Subject of Research: Bridge funding for academic therapeutic development programs in brain health, pediatrics and rare diseases

Article Title: C-Path’s Translational Therapeutics Accelerator awards $1.7 million across five bridge grants in brain health, pediatrics and rare diseases

Article References: C-Path’s Translational Therapeutics Accelerator awards $1.7 million across five bridge grants in brain health, pediatrics and rare diseases. (n.d.). Original publication

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Keywords: Critical Path Institute, BRIDGe grants, drug development, rare diseases, gene therapy, Usher Syndrome Type 1C, cryptosporidiosis, neuropathic pain, Gaucher disease, sepsis, blood-brain barrier, pediatrics