CHARLESTOWN, Mass. - Solid Biosciences Inc. (NASDAQ:SLDB) announced today that the U.S. Food and Drug Administration (FDA) has approved its Investigational New Drug (IND) application for SGT-212, a gene therapy candidate for the treatment of Friedreich’s ataxia (FA). This progressive disease is characterized by the body's inadequate production of the protein frataxin, leading to nervous system damage and cardiac dysfunction.
SGT-212 is a novel therapy using Adeno-Associated Virus (AAV) to deliver full-length frataxin directly to the cerebellum and heart. This dual administration approach is designed to address the neurological and cardiac symptoms associated with FA, potentially halting disease progression. According to InvestingPro data, the company maintains a strong liquidity position with a current ratio of 7.85, though analysts note it's quickly burning through cash reserves. Solid Biosciences' President and CEO, Bo Cumbo, emphasized the therapy's targeted delivery to the dentate nuclei and cardiac tissue, which has shown promising preclinical results, including safe transduction, frataxin expression, and significant restoration of function in mice.
Jennifer Farmer, CEO of the Friedreich’s Ataxia Research Alliance (FARA), recognized the milestone, highlighting the unmet medical needs within the FA community. FARA's partnership with Solid Biosciences aims to bring SGT-212 into clinical trials later this year, with the hope of addressing the debilitating neurological symptoms and life-shortening cardiac disease faced by those with FA.
Solid Biosciences plans to initiate a Phase 1b clinical trial in the second half of 2025 to assess the safety and tolerability of SGT-212 in both non-ambulatory and ambulatory adult FA patients. The trial will monitor participants for up to five years post-treatment.
The company will discuss the IND clearance and other corporate updates in a conference call scheduled for tomorrow at 8:30 AM ET. The call will be available via webcast on Solid Biosciences’ website and accessible by phone.
This announcement is based on a press release statement from Solid Biosciences Inc.
In other recent news, Solid Biosciences, a gene therapy company, has been the subject of positive coverage by JMP Securities and has entered into a significant collaboration with Mayo Clinic. JMP Securities initiated coverage on Solid Biosciences with a Market Outperform rating, noting the company's potential in gene therapy and its valuable capsid engineering capabilities. The firm's valuation of the company is justified by several factors, including a strong liquidity position and promising clinical-stage DMD asset.
In addition, Solid Biosciences has secured an exclusive collaboration with Mayo Clinic to develop gene therapies targeting genetic cardiac conditions. The agreement grants Solid Biosciences exclusive worldwide licenses to Mayo Clinic's Suppression-Replacement gene therapy platform and several cardiac gene therapy programs. This partnership aims to leverage Solid's advanced AAV capsids and manufacturing capabilities in conjunction with Mayo Clinic's platform to address life-threatening genetic heart diseases.
These are recent developments that highlight Solid Biosciences' ongoing efforts to develop and potentially commercialize its gene therapy technologies. As the company continues to advance in these areas, analysts suggest a favorable future for Solid Biosciences, particularly in light of its partnership with Mayo Clinic and its potential in cardiac precision genetic medicine.
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