NEW YORK, June 30, 2021 (GLOBE NEWSWIRE) — LEXEO Therapeutics, a clinical-stage gene remedy firm, in the present day introduced that the U.S. Meals and Drug Administration (FDA) has granted Uncommon Pediatric Illness designation and Orphan Drug designation to LX2006 for the remedy of Friedreich’s ataxia (FA). LX2006 is an IV-administered, adeno-associated virus (AAV)-mediated gene remedy encoding the human frataxin gene. The designations granted to LX2006 cowl cardiac illness and broader signs related to FA.
The FDA grants Uncommon Pediatric Illness designation for severe and life-threatening illnesses that primarily have an effect on youngsters ages 18 years or youthful and fewer than 200,000 folks within the U.S. If a biologics license software (BLA) for LX2006 is authorised, LEXEO could also be eligible to obtain a precedence assessment voucher which may be bought or transferred.
The Orphan Drug designation is granted by the FDA to medicine or biologics meant to deal with a uncommon illness that impacts fewer than 200,000 folks within the U.S. Applications with Orphan Drug standing are eligible for varied growth incentives.
“Being granted each Uncommon Pediatric Illness and Orphan Drug designation exhibits the large urgency for brand new, impactful therapeutic approaches akin to LX2006 for folks recognized with Friedreich’s ataxia,” mentioned R. Nolan Townsend, Chief Govt Officer of LEXEO Therapeutics. “It’s crucial that we advance new disease-modifying therapies with the potential to rework the lives of FA sufferers, and we stay up for persevering with our collaboration with the FDA as we advance LX2006 by means of scientific growth.”
FA is a uncommon, degenerative, multi-system dysfunction affecting roughly one in 50,000 folks in the USA. It’s an inherited situation brought on by a gene mutation that disrupts the conventional manufacturing of the protein frataxin, which capabilities within the mitochondria of the cell. It’s inherited in an autosomal recessive method, normally starting in childhood and results in impaired muscle coordination (ataxia) that worsens over time, sometimes progressing to severe coronary heart circumstances that may result in coronary heart failure, which is the commonest reason for demise in FA sufferers.
LEXEO plans to provoke a Part I/II scientific trial of LX2006 in sufferers with cardiomyopathy related to FA in 2021.
About LEXEO Therapeutics, Inc.
LEXEO Therapeutics is a New York Metropolis-based, absolutely built-in biotechnology firm at present headquartered on the Alexandria Middle® for Life Science that goals to use the transformational science of gene remedy to handle a few of the world’s most devastating genetic and purchased illnesses. LEXEO Therapeutics’ pipeline consists of adeno-associated virus (AAV)-mediated therapies primarily developed at Weill Cornell Drugs’s Division of Genetic Drugs. Past LEXEO Therapeutics’ lead packages – that are targeted on each uncommon and non-rare monogenic (single gene mutation) illnesses – the corporate’s preclinical pipeline spans monogenic illnesses, in addition to hereditary and purchased illnesses throughout a spectrum of affected person inhabitants sizes and a spread of unmet medical wants. Importantly, LEXEO Therapeutics will concentrate on advancing scientific packages by means of to commercialization, with the objective of sustaining an ongoing analysis collaboration with Weill Cornell Drugs’s Division of Genetic Drugs to assist advance the corporate’s pre-clinical pipeline. For extra info, please go to http://www.lexeotx.com/ or LinkedIn.
LEXEO Therapeutics, Inc.
Sheryl Seapy, Actual Chemistry