The CMT Research Foundation (CMTRF), a non-profit focused solely on delivering treatments and cures for Charcot-Marie-Tooth (CMT) disease*, today announced a partnership with Nanite, Inc., a Boston-based biotechnology company developing SAYER™, an AI-driven gene delivery platform focused on polymer nanoparticles (PNPs). As part of the partnership agreement, Nanite will design delivery vehicles for genetic medicines to Schwann cells via PNPs, an emerging technology for drug delivery to hard-to-hit targets.
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Approximately two-thirds of the world’s more than 3 million CMT cases are caused by mutations in genes that are expressed solely or almost solely within myelinating Schwann cells, including CMT1A, CMT1B, and CMT1X, among others. This makes Schwann cells the primary target of genetic therapies in most cases of CMT. However, myelinating Schwann cells are “locked away” behind the blood-nerve barrier (BNB), making the delivery of genetic medicines to Schwann cells a major challenge. The safe delivery of medicines to the Schwann cells of patients with CMT1A, would mark a dramatic step forward in the potential of gene therapies for CMT disease.
“A major step forward in the potential treatment of CMT1A was made in 2017 when researchers developed antisense oligonucleotides (ASOs) capable of decreasing PMP22 expression in rats and mice. However, because very little made it past the BNB, the ASOs had to be given at concentrations so high that they caused toxicity,” said Cleary Simpson, CEO of the CMTRF. “Our partnership with Nanite will use their SAYER platform to design PNPs to improve targeting of ASOs to Schwann cells, thereby lowering the effective dose to a point that toxicity is no longer a concern.”
“To ensure optimal delivery of ASOs to Schwann cells, PNPs will initially be designed and tested with Schwann cells in a dish to discover and optimize features that promote Schwann cell entry and genetic payload delivery, confirmed by PMP22 reduction,” said Shashi Murthy, Ph.D., Co-Founder and Chief Technology Officer of Nanite. “In the second phase of this partnership, Nanite will use the resulting data and harness the closed-loop optimization capability of SAYER to achieve ASO delivery to Schwann cells in vivo.”
The program is one of several building blocks directed towards validating Nanite’s platform as an important addition to the choices available to gene therapy companies.
Nanite Inc. is a next-generation non-viral gene delivery company developing a new class of programmable polymer nanoparticles for a range of modalities and indications. The company’s AI-driven platform, SAYER™, combines cutting-edge high-throughput experimental and computational methods to design fit-for-purpose delivery vehicles delivering a broad range of genetic cargoes with tissue specificity. Nanite is headquartered in Boston, MA. Follow us on Twitter @nanitebio.
The CMT Research Foundation (CMTRF) is focused solely on delivering treatments and cures for CMT. Founded by two patients who are driven to expedite drug delivery to people who live with CMT globally, the organization funds research for drug development. The 501(c)(3) federal tax-exempt organization is supported by personal and corporate financial gifts.
*Charcot-Marie-Tooth encompasses a group of inherited, chronic peripheral neuropathies that result in nerve degradation. CMT patients suffer from progressive muscle atrophy of legs and arms causing walking, running and balance problems and abnormal hand and foot functioning. CMT affects one in 2,500 people (about the same prevalence as multiple sclerosis), including 150,000 Americans and nearly 3 million people worldwide. At the moment, there is no treatment or cure for CMT.
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