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 mito 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 Mito research.

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 Mito CMTs.

Our most recent TRIAD -MITO partnerships & initiatives

Click on the logos for more information.

Rarebase – Mito
Rarebase leverages cutting-edge technology and biology using repurposed FDA-approved drugs to deliver accelerated, off-label treatments for MTRFR/C12orf65.

Click on the logos for more information.

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

Click on the logos for more information.

University of Cambridge – Mito
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 – Mito
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.
Envigo – Mito
A premiere laboratory animal sourcer, has developed a rat model for two types of CMT - CMT4A-GDAP1 and SORD deficiency
Jackson Labs – Mito
The Burgess Mouse Laboratory is a state-of-the-art facility working to develop mouse models for Mito CMTs, including the MTRFR/C12orf65 gene to test repurposed drugs and gene therapies targeting the optic nerve and other phenotypes of disease.

Click on the logos for more information.

University of Helsinki – Mito
The Battersby Lab receives support from HNF to develop an effective treatment approach for C12orf65 deficiency (also known as MTRFR).
Veneto – Mito
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 Mito CMTs 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

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

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”).

Pharnext Announces Pleotherapy Proof of Concept in Charcot-Marie-Tooth Disease Type 1A

Pharnext Announces Pleotherapy Proof of Concept in Charcot-Marie-Tooth Disease Type 1A

PARIS, December 18th, 2014 – Pharnext SAS today announced the proof of concept of its pleotherapy research and development approach based on a proprietary network pharmacology platform that identifies synergic combinations of drugs already approved for other diseases. Indeed, Pharnext’s lead pleodrug, PXT-3003, has shown positive results both in preclinical and Phase 2 clinical studies published today in the Orphanet Journal of Rare Diseases.