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US FDA clears Solid Biosciences’ IND application for SGT-212 to treat neurologic & cardiac manifestations of Friedreich’s ataxia

Charlestown, Massachusetts
Thursday, January 9, 2025, 14:30 Hrs  [IST]

Solid Biosciences Inc, a life sciences company developing precision genetic medicines for neuromuscular and cardiac diseases, announced that the US Food and Drug Administration (FDA) has cleared its investigational new drug (IND) application for SGT-212 for the treatment of Friedreich’s ataxia (FA), a degenerative disease caused by insufficient levels of the frataxin protein.

SGT-212 is the only full-length frataxin replacement gene therapy candidate targeting the CNS and cardiac manifestations of Friedreich’s ataxia.  SGT-212 is the company’s novel, AAV-based Friedreich’s ataxia gene therapy candidate designed to deliver full-length frataxin via systemic intravenous (IV) infusion as well as direct intradentate nuclei (IDN) infusion into the cerebellum. SGT-212 is designed to treat the neurologic and systemic clinical manifestations of Friedreich’s ataxia to address the full spectrum of disease progression.

Friedreich’s ataxia is a highly complex, multisystem disease that presents distinct challenges for drug development, in part because frataxin is a protein that requires:  precise expression levels to avoid fatal cardiac toxicities, and on-target tissue localization in the cerebellum to achieve potential neurological clinical benefit.

SGT-212 is the only candidate in development using two routes of administration to address the cardiac manifestations of Friedreich’s ataxia while also directly delivering therapy to the dentate nuclei in the cerebellum, the region most affected and implicated in FA-associated neurologic decline.

“SGT-212 has been intentionally designed to enable highly targeted delivery of our gene therapy to both the dentate nuclei and cardiac tissue. The IND was supported by a robust preclinical package demonstrating safe transduction and frataxin expression in these target tissues, with significant restoration of neurologic function and reversal of the cardiac implications of the disorder in mice. Over the years, we have tested several candidates using different methods of administration and have conducted multiple NHP studies, some of which extended out to a year. Based on this research, we believe a dual route of administration targeting multiple systems is the best approach in development to directly address the neurological implications that profoundly impact the everyday life of patients, while simultaneously targeting the cardiac manifestations that play a key role in more progressed disease. SGT-212 offers a truly differentiated approach to addressing FA with the potential to treat the full spectrum of symptoms, and we hope to meet each patient where they are in their FA disease course”, said  Bo Cumbo, president and CEO of Solid Biosciences.

“We congratulate Solid Biosciences on reaching this significant milestone. Gene therapy approaches are aimed at the underlying causes of FA, and thus important in the overall strategy to treat and cure this disease. There has been encouraging progress in the FA treatment landscape; however, there is still unmet medical need for our patient community. Through our work with individuals living with FA and their families, we know they seek therapies designed to treat the debilitating neurologic symptoms that people living with FA face day-to-day, such as loss of ambulation and coordination, dysarthria, along with the life-shortening cardiac disease. SGT-212’s unique, precision approach targets both the cerebellum and cardiac tissue using a dual route of administration, and in doing so, aims to address the underlying cause of the disease and the progression of FA. We look forward to continued partnership with the Company as they advance SGT-212 into the clinic later this year,” added Jennifer Farmer, CEO of the Friedreich’s Ataxia Research Alliance.

In the second half of 2025, the Company expects to initiate a first-in-human, open-label, dose-finding phase 1b clinical trial of SGT-212. The study will enroll non-ambulatory and ambulatory adult patients living with FA across up to three cohorts and will evaluate the safety and tolerability of contemporaneous systemic and bilateral IDN administration of SGT-212. Participants in the trial will be followed out to five years after receiving SGT-212.

SGT-212 is a recombinant AAV-based gene replacement therapy for Friedreich’s ataxia designed to deliver full-length human frataxin (Fxn) via a dual route of administration: intradentate nucleus (IDN) infusion, using an MRI-guided device, followed by an intravenous (IV) infusion to increase therapeutic Fxn levels in the cerebellar dentate nuclei and in the cardiomyocytes, respectively. Restoration of Fxn levels is expected to repair the underlying mitochondrial dysfunction in neurons and cardiomyocytes to address both neurologic and cardiac manifestations of the disease.

 

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