Impact That Matters

Therapeutic Research In Accelerated Discovery (TRIAD)
 

TRIAD = Academia + Government + Industry

TRIAD Partners

Our TRIAD partners include innovative biotech & pharmaceutical companies, award-winning researchers at top-tier universities, and those spanning the drug discovery, drug development, and diagnostics continuum for Charcot-Marie-Tooth research.

Partnerships vary depending on research goals, milestones met in preclinical or clinical development, and strategic alliance agreements.

TRIAD & GRIN

As part of TRIAD, in 2013, the Global Registry for Inherited Neuropathies (GRIN) was developed to conduct patient-focused research & development for treatments and cures. The patient voice is at the forefront of all we do. By incorporating the patient voice from the beginning, our research programs have the greatest potential for success. HNF funds research with one goal in mind; advancing to clinical trials for Charcot-Marie-Tooth research.

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TRIAD Council

The TRIAD Council is composed of CMT thought leaders, experts, and consultants engaged in collaborative planning and decision-making to provide regular guidance and direction to our research strategy. This network of professionals reviews grant proposals, provides expert guidance, and assesses project outcomes to advance therapeutic development for CMT.

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Our most recent TRIAD partnerships & initiatives

Click on the logos for more information.

Rarebase
Leverages cutting-edge technology and biology using repurposed FDA-approved drugs to deliver accelerated, off-label treatments for various types of CMT.
Miralinc Pharma
Pioneering Next Generation HDAC6i’s to advance life-changing disease cures for CMT patients and other peripheral neuropathies.
Pharnext Pharmaceuticals
Clinical-stage biopharmaceutical company, partnered with HNF in 2012 to support the development of PXT3003. the protein impacted in CMT1A.
Applied Therapeutics
Clinical-stage biopharmaceutical company conducting a trial for patients with Sorbitol Dehydrogenase (SORD) Deficiency.

Click on the logos for more information.

Genome Medical
Nationwide medical practice specializing in virtual genetic counseling services.
Matrix by Across HealthCare
In 2022, HNF re-launched its Global Registry for Inherited Neuropathies (GRIN) on the Matrix platform by Across Healthcare.
Vitaccess / CMT&Me App
International study to collect real-time patient data directly to determine how CMT impacts patients’ quality of life.

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University of Cambridge
One of the top academic centers in the world, conducting a retrospective natural history study for CMT6, the C12orf65 (also referred to as MTRFR) gene.
Burke Institute
The Willis Lab has developed a CMT4A rat model developed and owned by HNF to test a gene therapy to treat GDAP1, a recessive CMT4 subtype.
University of Miami
The Saporta CMT Stem Cell Laboratory is a state-of-the-art facility working to develop iPSc’s for CMT4A (GDAP1) and CMT6 (C12orf65, also known as MTRFR gene).
The Genesis Project
A genomic data information system platform for real-time sharing of CMT genome-level data to advance research.
University of San Antonio Texas
Principal Investigator Manzoor Bhat, PhD has dedicated his career to the understanding of a rare and fatal type of Inherited Neuropathy called CNTNAP1.
Envigo
A premiere laboratory animal sourcer, has developed a rat model for two types of CMT - CMT4A-GDAP1 and SORD deficiency
Wuxi Biologics
Supporting PK dog studies to advance the HDAC6i program for CMT2A.

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University of Helsinki
The Battersby Lab receives support from HNF to develop an effective treatment approach for C12orf65 deficiency (also known as MTRFR).
Veneto Institute of Molecular Medicine – V.I.M.M.
A gift was awarded to support “an unbiased screening of FDA approved drugs to identify small molecules counteracting axonal mitochondrial depletion in Autosomal Dominant Optic Atrophy (OPA1 gene) RGCs.

HNF participates and supports an advocacy role by providing real-world data to the FDA and other stakeholders. Our critical data facilitates improved knowledge of the lived experience of CMT patients and the advancement of clinical development.

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Latest News and Related Blog Posts

Pharnext Announces PXT3003 for the Treatment of Charcot-Marie-Tooth Disease Type 1A has Been Granted Promising Innovative Medicine (PIM) Designation by UK Medicines and Healthcare Products Regulatory Agency

by | Mar 18, 2020 | , | 5 Comments

United Kingdom’s Medicine and Healthcare products Regulatory Agency (MHRA) has granted Promising Innovative Medicine (PIM) designation to its lead drug candidate, PXT3003, for the treatment of Charcot-Marie-Tooth Disease Type 1A (CMT1A) in patients 16 years and older.

Pharnext raises € 7.7 million in a private placement

by | Mar 11, 2020 | , , | 0 Comments

Pharnext SA (FR0011191287 – ALPHA) (the “Company”), a biopharmaceutical company pioneering a new approach to developing innovative drug combinations based on its PLEOTHERAPY artificial intelligence platform harnessing big genomics data and network pharmacology, today announced a capital raise of circa € 7.7 million by way of issuance of 1,799,061 new ordinary shares (the “New Shares”) with one warrant attached each (together with the New Shares, the “ABSA”).