Five research teams advance small molecule, protein-based and gene therapy candidates for conditions where existing options fall short for those most affected
TUCSON, Ariz., September 15, 2026 — Critical Path Institute® (C-Path) today announced that its Translational Therapeutics Accelerator has awarded $1,699,500 through five Bridging Research and Innovation in Drug Development Grants (BRIDGe). Announced individually between March and July 2026, the projects address sepsis-related cognitive decline, inherited blindness, neurological lysosomal storage disease, pediatric cryptosporidiosis, and neuropathic pain.
All five awards fall within the accelerator’s three priority areas: brain health, pediatrics, and rare and orphan diseases. Together they cover every modality the program funds, with three small molecule programs, one protein-based therapeutic and one in vivo gene therapy.
The awards are:
- $242,781 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, to develop sigma-1 receptor antagonists as nonopioid treatments for neuropathic pain and chemotherapy-induced peripheral neuropathy. The team is building a pharmacokinetic/pharmacodynamic model to guide compound.
- $249,719 to Odylia Therapeutics, a nonprofit biotechnology organization in Atlanta led by Ashley Winslow, Ph.D., for an adeno-associated virus gene therapy that delivers a functional copy of the USH1C gene to retinal cells. Children with Usher Syndrome Type 1C can receive cochlear implants for hearing loss. Nothing currently prevents or reverses the retinal degeneration that follows in adolescence.
- $250,000 to Teresa Sanchez, Ph.D., at Weill Cornell Medicine, for an antibody-based therapy that targets a vascular mechanism involved in maintaining the blood-brain barrier. 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 no effective treatment.
- $456,000 to James Shayman, M.D., at the University of Michigan, for BPN-25271, a brain-penetrant glucosylceramide synthase inhibitor developed with medicinal chemist Scott Larsen, Ph.D. Dr. Shayman previously invented eliglustat tartrate, the only oral therapy approved globally for Gaucher disease type 1. Because eliglustat does not cross the blood-brain barrier, it leaves untreated the neurological progression seen in Gaucher disease type 3, GM1 gangliosidosis, Tay-Sachs disease and Sandhoff disease.
- $501,000 to Wesley Van Voorhis, M.D., Ph.D., at the University of Washington School of Medicine, to advance BKI-1708, a selective CpCDPK1 kinase inhibitor, toward an Investigational New Drug application for cryptosporidiosis. C-Path has reported that the disease causes an estimated 200,000 deaths each year, concentrated among children under five in low- and middle-income countries, and that the one marketed therapy has limited efficacy in malnourished children.
“Every promising result we see in the lab is just the first page of a much larger story,” said Maaike Everts, Ph.D., Executive Director of C-Path’s Translational Therapeutics Accelerator. For these five teams, the science has reached a critical point where the right resources and expertise can help determine what comes next. BRIDGe grants provide that momentum, 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.”
BRIDGe awards are structured around clear milestones and go/no-go decision points, aligning the funding directly with the project’s specific work plan.
Awardees retain complete ownership of their intellectual property and gain a dedicated support system to advance their research. 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. To keep the science moving efficiently, C-Path also offers flexible financial execution. If a study requires external capabilities, C-Path helps investigators find the right contract research organizations or consultants and allows a substantial portion of the grant to fund those partners directly.
“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. “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, we can help innovators anticipate what will be needed and generate the right evidence at the right time.”
C-Path’s Translational Therapeutics Accelerator funds faculty at universities and nonprofit institutions anywhere in the world, across small molecules, protein-based therapeutics and in vivo gene-based therapies. Award sizes 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 (PKD) in partnership with the PKD Foundation, the first collaboration of its kind for the program. It builds 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.
TRxA closed its 2026 BRIDGe funding cycle to full proposals on August 12, 2026, with additional awards to be announced following review.
To learn more, visit c-path.org/programs/trxa or contact the team at trxa@c-path.org.
About Critical Path Institute
Critical Path Institute (C-Path) is an independent, nonprofit established in 2005 as a public-private partnership, in response to the FDA’s Critical Path Initiative. C-Path’s mission is to lead collaborations that advance better treatments for people worldwide. Globally recognized as a pioneer in accelerating drug development, C-Path has established numerous international consortia, programs and initiatives that currently include more than 1,600 scientists and representatives from government and regulatory agencies, academia, patient organizations, disease foundations and pharmaceutical and biotech companies. With dedicated team members located throughout the world, C-Path’s global headquarters is located in Tucson, Arizona and C-Path’s Europe subsidiary is headquartered in Amsterdam, Netherlands. For more information, visit c-path.org.
About C-Path’s Translational Therapeutics Accelerator
Critical Path Institute’s Translational Therapeutics Accelerator is a global drug accelerator focused on supporting academic scientists in advancing novel therapeutics from university-based labs to drug development pipelines of pharmaceutical companies and, ultimately, the clinic. As a nonprofit neutral convener of patient groups, academia, pharmaceutical companies and regulatory agencies, C-Path brings a breadth of scientific and drug development planning not available in other accelerator programs. C-Path’s accelerator is uniquely situated to leverage the expertise available through C-Path’s >20 disease-based consortia, as well as regulatory expertise and project management, to empower academic investigators to succeed in bringing safe and effective treatments to patients. For more information, visit c-path.org/trxa or email trxa@c-path.org.
Media Contacts:
Roxan Triolo Olivas
C-Path
520.954.1634
rolivas@c-path.org
Kissy Black
C-Path
615.310.1894
kblack@c-path.org
