SAN RAFAEL, Calif., Oct. 18, 2017 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) updated the investment community on the Company's development portfolio, which is focused on innovative therapies to treat rare and ultra-rare diseases.
BioMarin announced today that it has selected BMN 290, a selective chromatin modulation therapy, for the treatment of Friedreich's Ataxia (FA). FA is a rare autosomal recessive disorder with worldwide prevalence of approximately 15,000, which results in disabling neurologic and cardiac progressive decline. Currently there are no approved disease modifying therapies for FA. In preclinical models, BMN 290 increases frataxin expression in affected tissues more than two-fold. BMN 290 is a second generation compound derived from a compound acquired from Repligen that had human clinical data demonstrating increases in frataxin in FA patients. BMN 290 was selected for its favorable penetration into the central nervous system and cardiac target tissues, and its preservation of the selectivity of the original Repligen compound. The company expects to submit the IND in 2H 2018.
Wednesday, October 18, 2017
New data for Friedreich's ataxia with a novel capsid demonstrate reversal of disease phenotype in a preclinical disease model.
Press Release: CAMBRIDGE, Mass., Oct. 17, 2017 (GLOBE NEWSWIRE) -- Voyager Therapeutics, Inc. (NASDAQ:VYGR), a clinical-stage gene therapy company focused on developing life-changing treatments for severe neurological diseases announced today multiple data presentations at the Congress of the European Society of Gene and Cell Therapy (ESGCT) taking place October 17-20, 2017, in Berlin, Germany. The data include an oral presentation related to recent results from Voyager's ongoing Phase 1b trial of VY-AADC01 in advanced Parkinson's disease, as well as six poster presentations related to Voyager's novel adeno-associated virus (AAV) capsid optimization efforts, gene therapy manufacturing, and preclinical pipeline programs.
Rescue of Central and Peripheral Neurological Phenotype in a Novel Mouse Model of Friedreich's Ataxia by Intravenous Delivery of AAV Frataxin." Poster P107.
Friedreich's ataxia is a severe, inherited neurological disease caused by mutations in the frataxin gene leading to decreased expression of frataxin (FXN), which results in severe sensory impairment, progressive loss of the ability to walk, generalized weakness, loss of sensation, as well as severe and potentially fatal cardiomyopathy. In a transgenic mouse model of FA, one-time intravenous post-symptomatic dosing of an AAV vector composed of a novel AAV capsid and a frataxin transgene, together with intracerebral dosing also delivering a frataxin transgene, rapidly halted and reduced FA disease progression in multiple tests including three functional tests of motor behavior and one electrophysiological test. In addition, increasing intravenous vector doses with the same novel capsid together with a fixed dose of the intracerebral vector led to a dose-dependent rescue of the FA phenotype. This novel AAV capsid provided at least 20-fold greater delivery of the vector to sensory ganglia as measured by vector genomes, and approximately a three-fold greater expression of frataxin in the cerebellum, as compared to an AAV9 vector at a similar dose. Additional preclinical studies are underway at Voyager including steps to optimize a lead clinical candidate for the treatment of FA.
New data for Friedreich's ataxia with a novel capsid demonstrate reversal of disease phenotype in a preclinical disease model.
Rescue of Central and Peripheral Neurological Phenotype in a Novel Mouse Model of Friedreich's Ataxia by Intravenous Delivery of AAV Frataxin." Poster P107.
Friedreich's ataxia is a severe, inherited neurological disease caused by mutations in the frataxin gene leading to decreased expression of frataxin (FXN), which results in severe sensory impairment, progressive loss of the ability to walk, generalized weakness, loss of sensation, as well as severe and potentially fatal cardiomyopathy. In a transgenic mouse model of FA, one-time intravenous post-symptomatic dosing of an AAV vector composed of a novel AAV capsid and a frataxin transgene, together with intracerebral dosing also delivering a frataxin transgene, rapidly halted and reduced FA disease progression in multiple tests including three functional tests of motor behavior and one electrophysiological test. In addition, increasing intravenous vector doses with the same novel capsid together with a fixed dose of the intracerebral vector led to a dose-dependent rescue of the FA phenotype. This novel AAV capsid provided at least 20-fold greater delivery of the vector to sensory ganglia as measured by vector genomes, and approximately a three-fold greater expression of frataxin in the cerebellum, as compared to an AAV9 vector at a similar dose. Additional preclinical studies are underway at Voyager including steps to optimize a lead clinical candidate for the treatment of FA.
New data for Friedreich's ataxia with a novel capsid demonstrate reversal of disease phenotype in a preclinical disease model.
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