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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