News | October 12, 2021

HNF Collaborates With Rarebase On A Drug Discovery Platform To Develop Treatments For Charcot-Marie-Tooth (CMT)

New York, NY /PRNewswire/ -- The Hereditary Neuropathy Foundation (HNF), in partnership with Rarebase, is leading the charge in the first-ever research initiative to tackle multiple types of CMT in one project. Rarebase is a public benefit biotech company focused on accelerating therapy development for rare diseases with its tech-enabled drug discovery platform called "Function". Rarebase will screen a compound library of thousands of FDA-approved drugs and novel drugs, targeting ten subtypes of Charcot-Marie-Tooth (CMT).

The primary goal of the collaboration is to identify the most promising drugs for each subtype of CMT and test these candidate drugs in patient-derived cellular models, specifically; Induced Pluripotent Stem Cell derived neurons (iPSC-neurons). These cell-based models will enable the collaboration to identify and characterize potential therapies more quickly and cost effectively and reduce our dependence on research animal studies. In addition, HNF's Patient-Driven Therapeutic Research In Accelerated Discovery (TRIAD) program will connect Rarebase with its CMT researchers, clinicians, and other stakeholders worldwide to potentially expand the research team.

To date, there are no approved treatments for any type of CMT. Most research is slow moving, siloed and often duplicative. The technological innovation and fast-paced scientific team at Rarebase will share preliminary results as early as the first quarter of 2022.

"We're thrilled to be collaborating with the HNF to help find treatments for patients with CMT. There are thousands of approved drugs, and many of them can have applications beyond their routinely prescribed indication. If such treatments are out there for CMT, we will find them," said Onno Faber, Co-Founder and CEO of Rarebase.

CMT is a debilitating and progressive disease impacting an estimated 150,000 people in the US and millions worldwide. Scientists have identified over 100 different gene mutations causing CMT. Currently, thousands of people with CMT are undiagnosed, yet are suffering with the symptoms of this chronic and disabling disease. CMT causes progressive nerve damage, eventually leading to muscle atrophy and loss. Over time the disease leads to feet, legs, and hands becoming deformed and difficult to use and often those living with CMT lose the ability to walk normally and become dependent on assistive devices to remain mobile. Severe, chronic pain is common.

"Our core mission is to deliver treatments to patients with CMT. We believe the Rarebase partnership will enable FDA approved drugs for clinical use for CMT. If successful, HNF plans to expand the screens to other subtypes as funds become available," said Allison Moore, the Founder and CEO of the Hereditary Neuropathy Foundation.

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About Hereditary Neuropathy Foundation (HNF)
HNF is a non-profit 501(c) 3 organization whose mission is to increase awareness and accurate diagnosis of CMT and related inherited neuropathies, support patients and families with critical information to improve quality of life, and fund research that will lead to treatments and cures. HNF developed the Therapeutic Research in Accelerated Discovery (TRIAD) program, a collaborative effort with academia, government and industry, to develop treatments for CMT. Currently, TRIAD involves many groups that span the drug discovery, drug development and diagnostics continuum. HNF encourages patients to join the Global Registry for Inherited Neuropathies (GRIN), a patient registry that helps to advance clinical research and enables patients to be contacted to participate in research pilot studies and potential clinical trials.

About Rarebase, PBC
Rarebase is a public benefit biotech led by patients, scientists, advocates and engineers. We leverage innovative technologies to build platforms that help us discover and develop treatments for the millions of people worldwide affected by the long tail of rare genetic disorders. The company is funded by BlueYard Capital.

Source: Hereditary Neuropathy Foundation

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