Peter Barton Hutt, a visionary leader in food and drug law, passed away on September 2, 2026. He was a co-author of the leading casebook and testified before Congress over 100 times, shaping modern regulatory science. C-Path mourns his loss and thanks him for his service to the organization and public health.
The publication of the alpha-synuclein seed amplification assay (αSyn-SAA) biomarker highlights a decade of collaborative progress in biomarker development. This milestone enables earlier testing of new treatments for Parkinson's disease, which could delay or prevent disease onset and progression.
Critical Path Institute (C-Path) has achieved ISO/IEC 27001:2022 certification, demonstrating its commitment to protecting sensitive information and partner trust. This certification reflects the organization's structured risk-based information security management system.
The coalition aims to facilitate innovative use of NAM tools, closing the gap in regulatory adoption. Crown Bioscience brings expertise in patient-derived models and biomarker analysis to support standardization of NAMs for regulatory acceptance.
Karen Bernstein, co-founder of BioCentury Inc., has been elected as the new chair of Critical Path Institute's (C-Path) Board of Directors. She succeeds M. Wainwright Fishburn, Jr. Bernstein brings her expertise in drug development and analysis to the role, having worked in the biopharmaceutical sector since 1987.
A new $250,000 grant will support the development of a novel therapeutic approach for sepsis-associated neurocognitive dysfunction. The treatment aims to protect the brain from inflammation-related damage during and after sepsis, potentially preventing or slowing neurodegeneration and cognitive decline.
The Critical Path Institute's Type 1 Diabetes Consortium is strengthening regulatory science and evidence frameworks for disease-modifying therapies. The expanded C-peptide evidence base supports ongoing evaluation of the biomarker as a drug development tool.
Dr. Milad Alucozai joins C-Path's Board of Directors with extensive experience in U.S. biotechnology and startup community, including founding and investing in companies like Pamir Ventures and Revalia Bio. His appointment reflects the organization's commitment to integrating emerging biotechnology into its global mission.
Yalonda Howze joins C-Path's Board of Directors with nearly two decades of experience in biotechnology and life-science sectors. Her appointment aims to accelerate drug development and advance therapies for rare diseases.
The conference will convene leaders from regulatory agencies, industry, academia, and advocacy to discuss establishing a new paradigm for evidence of efficacy and safety. The agenda features sessions on artificial intelligence, real-world data, digital health technologies, and trust in data sharing.
The coalition aims to accelerate validation and qualification of human-relevant methodologies, such as complex in vitro models and microphysiological systems, for drug discovery and development. By aligning developers around shared qualification standards, the coalition reduces duplicative validation efforts and helps regulators evalua...
Odylia Therapeutics is advancing a gene therapy treatment for Usher Syndrome Type 1C (USH1C)-associated vision loss, a rare autosomal recessive disorder. The grant will support the development of an investigational USH1C gene therapy designed to restore or preserve vision.
A $456,000 grant from Critical Path Institute will support the development of a brain-penetrant therapy for glycosphingolipid storage disorders. The therapy aims to address neurological symptoms in patients with current limited treatment options.
The consortium aims to forge actionable drug development solutions for patients living with AATD by collaborating on endpoints, biomarkers, and clinical trial design. AIRNA brings RNA editing expertise to the consortium's efforts to accelerate drug development for alpha-1 antitrypsin deficiency.
The Critical Path Institute has launched a global, multi-stakeholder initiative to enable scalable development of advanced therapies for small patient populations. The 'One to Millions' program advances regulatory frameworks and leverages data platforms to support evidence generation and development pathways.
A new study found that functional impairment worsened over time in individuals with early Parkinson's disease, despite plateaued symptoms, with gait, balance, and posture being the most bothersome issues. The study's results underscore the importance of incorporating real-world measures of disease progression in clinical trial design.
The Critical Path Institute's Translational Therapeutics Accelerator is offering grants to support the development of new treatments for polycystic kidney disease, brain health, pediatrics, and rare diseases. C-Path has partnered with the PKD Foundation to offer a specialized track focused on PKD, and is also accepting applications fro...
The Critical Path Disease Modeling Coalition (CP-DMC) aims to develop regulatory-grade, quantitative disease progression models for pediatric Crohn's disease and ulcerative colitis. These models will transform clinical trial data into actionable descriptions of disease trajectories and treatment response.
The Critical Path Institute has been recognized for its innovative approaches to regulatory science, which have measurably improved public health. The organization's work in creating practical tools and cross-sector collaborations has shortened drug development timelines and improved regulatory decisions.
Colin Hill joins C-Path's Board of Directors with experience in AI, drug development, and translational acceleration. He aims to bring precision medicine focus to advance treatments worldwide.
The conference focused on accelerating drug development for rare diseases, neurology, type-1 diabetes, and pediatrics, with a focus on patient-centered evidence generation. Key commitments included early interception, trial modernization, and patient-first approaches.
The C-Path Translational Therapeutics Accelerator has completed a record year of funding and mentorship, supporting seven projects in 2025 with approximately $2.48 million in awards. The program awarded grants to teams advancing treatments for rare liver disease, drug-resistant lung infections and pediatric brain tumors.
The 2025 Critical Path Institute Global Impact Conference will bring together industry, regulatory agencies, academia, and patient advocacy groups to discuss pressing challenges in drug development. The event aims to foster cross-sector collaboration to advance medical innovation and improve treatment outcomes for patients.
Critical Path Institute's Translational Therapeutics Accelerator awards $250,000 to researchers advancing a promising therapeutic pathway for type 1 diabetes. The project aims to address key challenges in T1D treatment, including beta-cell preservation and immune system modulation.
The C-Path-led initiative brought together clinical trial sponsors, eCOA technology providers, and regulators to identify and address eCOA implementation issues. Key resources developed during the initiative include guidance on COA data collection and a more open, collaborative environment.
Researchers have identified six promising biomarkers that can detect drug-induced kidney injury earlier than traditional markers, enabling more timely monitoring during drug development. These biomarkers could lead to the development of safer medications and improve patient outcomes.
Researchers propose GLDH as a more liver-specific biomarker for detecting liver injury, offering greater specificity than current ALT and AST tests. The test performed similarly to ALT for detecting liver injury but was not impacted by associated muscle injury.
Critical Path Institute's Translational Therapeutics Accelerator (TRxA) is launching its 2025 global Request for Proposals to support academic researchers in bridging the 'valley of death' in drug development. The program aims to enable the transition of innovative therapeutics from lab to patients.
A new research grant aims to develop kinase inhibitors for treating type 1 diabetes, with a focus on preventing and reversing the condition. The grant is made through C-Path's TRxA program in partnership with The Leona M. and Harry B. Helmsley Charitable Trust.
The C-Path initiative aims to generate drug development solutions that reflect each person's unique experience, leveraging diverse data and innovative technologies like AI and digital health. GEM-PD seeks to address unmet need in Parkinson's disease therapies by focusing on sex and gender differences.
Critical Path Institute (C-Path) has announced key leadership appointments: Dr. Diane Stephenson promoted to Vice President of Neurology and Nadine Tatton welcomed as new Executive Director of CPAD Consortium. This move aims to advance neurodegenerative disease research, including Alzheimer's and Parkinson's diseases.
The FDA Letter of Support recognizes the potential of αSyn-SAA to accelerate clinical trial design in Parkinson's and related disorders. This biomarker can objectively detect early biology related to these diseases, enabling research toward tailored therapies and potentially delaying or preventing disease onset.
A new task force formed under C-Path's Rare Disease Cures Accelerator-Data and Analytics Platform aims to advance therapeutic development for Progressive Supranuclear Palsy (PSP). The collaboration leverages collective expertise to foster innovation, identify critical gaps in research, and expedite treatment advancements.
A new task force launched by C-Path aims to accelerate drug development for mitochondrial and inherited metabolic diseases. The task force will leverage C-Path's expertise in data management standards, biomarkers, and regulatory science to generate solutions and contribute to regulatory decision-making.
The C-Path Neuroscience Annual Workshop brought together stakeholders to chart a transformative course for neurology research and drug development, focusing on chronic progressive diseases such as Alzheimer's and Parkinson's. Key highlights included recommendations for innovative therapies and tools to address complex disorders.
Research leaders collaborate with organizations and regulators to develop digital tools for Parkinson's clinical trials. The Digital Drug Development Tools initiative accelerates the regulatory endorsement of these tools, enabling more objective and patient-centered measures.
Academic scientists will receive funding to establish a pleiotropic brain-penetrant small-molecule to impede glioblastoma and develop an epigenetic therapy for Prader-Willi syndrome. The TRxA program aims to bridge the drug development 'valley of death' and advance novel therapeutics from lab to clinical trials.
The FDA has issued a Biomarker Letter of Support for four novel biomarkers identifying drug-induced pancreatic injury. These microRNAs will enhance the detection of acute drug-induced pancreatic injury (DIPI) in phase 1 clinical trials, improving safety and accuracy.
The Critical Path Institute has launched a pre-consortium collaboration with the FDA's CBER and CDER to accelerate medical product development in alpha-1 antitrypsin deficiency. The collaboration aims to define unmet needs in drug development and lay the groundwork for specific solutions.
The Critical Path Institute (C-Path) has established a public-private partnership with the FDA and NIH to advance treatments for rare neurodegenerative diseases. The partnership will leverage C-Path's expertise in data management, quantitative analytics, and regulatory science to accelerate medical product development.
The collaboration aims to accelerate addressing unmet needs in the neonatal population by applying quantitative modeling, biomarkers, and regulatory science. Patients and families will benefit from improved understanding of bronchopulmonary dysplasia and better patient enrollment for trials.
The collaboration aims to create an interoperable global data ecosystem for rare diseases, accelerating the development of new therapies. This partnership benefits patients, regulators, advocacy stakeholders, researchers, and industry, while expanding C-Path's global capabilities in collaborating on methodologies and technologies.
The Rare Disease COA Consortium aims to optimize COA selection during medical product development for rare diseases. The consortium will identify and describe potentially fit-for-purpose publicly available COAs, reducing the time and cost associated with developing new ones.
A new analytics platform, RDCA-DAP, will be launched to accelerate rare disease treatment innovation by hosting and standardizing rare diseases data. The platform is expected to empower patients and families to drive innovation in the field.
The UNITE4TB partnership aims to develop new and highly active TB treatment regimens for drug-resistant and -sensitive TB. The 7-year project will utilize advanced clinical trials networks, adaptive trial designs, and machine learning techniques to accelerate the development of better treatment solutions.
The C-Path Virtual Workshop will focus on implementing endpoints and outcome measures for clinical trials in new-onset type 1 diabetes. Over 300 attendees, including researchers, pharmaceutical representatives, and regulatory experts, are expected to participate in the two-day virtual event.
C-Path and Tufts Medical Center collaborate to integrate patient-level clinical trial data and real-world data to generate actionable real-world evidence for neonatal drug development. The partnership aims to improve survival and outcome for preterm neonates with limited new drug development.
The Duchenne Regulatory Science Consortium (D-RSC) database will share individual-level data with qualified researchers to accelerate therapy development for Duchenne muscular dystrophy. The database includes data from clinical trials and natural history studies, and is fully de-identified to ensure participant anonymity.
The expanded patient-level data will facilitate more efficient clinical trial design, enabling generation of novel drug development tools and solutions. The novel tools and solutions will enable better informed inclusion criteria, endpoint selection, and patient enrichment strategies.
The Acute Kidney Injury Project aims to develop improved predictive and detection tools for kidney injury, addressing a global public health concern. The project will focus on developing prognostic tools to identify drug-induced kidney injury, with the goal of improving patient safety during clinical trials and advancing drug development.