LEXEO Therapeutics Launches $ 85 Million Series A Funding to Develop Gene Therapies for Rare and Non-Rare Monogenic Diseases
Rare disease and gene therapy industry veterans Steven Altschuler, MD R. Nolan Townsend and Jay Barth, MD partner with gene therapy pioneer Ronald Crystal, MD to launch gene therapy company fully integrated
Financing conducted, structured and syndicated by Longitude Capital and Omega Funds
The full pipeline includes three clinical stage gene therapy programs in monogenic diseases and up to 15 potential additional AAV gene therapy programs in monogenic and acquired diseases primarily developed at Weill Cornell Medicine
NEW YORK, Jan. 07, 2021 (GLOBE NEWSWIRE) – LEXEO Therapeutics, a clinical-stage gene therapy company, debuted today with $ 85 million oversubscribed Series A funding, led by Longitude Capital and Omega Funds, and joined by Lundbeckfonden Ventures, PBM Capital, Janus Henderson Investors, Invus, Woodline Partners LP, Alzheimer’s Drug Discovery FoundationI and Alexandria Venture Investments. Proceeds from the funding will help advance the Company’s three main investigative programs, including: LX2006, an intravenous treatment for cardiomyopathy associated with Friedreich’s ataxia (phase 1 start scheduled for 2021); LX1004, a CNS administered therapy for CLN2 Batten disease (phase 1/2 completed); and LX1001, a CNS-administered therapy for Alzheimer’s disease associated with APOE4 (phase 1 ongoing).
“We are delighted to launch today the mission to advance LEXEO’s promising pipeline of clinical-stage gene therapy treatments for patients diagnosed with some of the most challenging diseases in society,” said R. Nolan Townsend, Managing Director of LEXEO Therapeutics. “We are pleased to collaborate with Dr. Ronald Crystal – one of the industry’s most accomplished pioneers in the field of gene therapy – and, building on my years of leadership experience in the field of gene therapy. rare diseases at Pfizer, together we will build a world-class gene therapy organization. guided by leading science that has the potential to address a range of therapeutic indications. “
Founder and Chief Scientific Advisor, Dr Ronald Crystal is Professor and Chairman of Weill Cornell’s Department of Genetic Medicine and Director of the Belfer Gene Therapy Platform. Dr Crystal has over 30 years of experience with adenoviruses and adeno-associated viral vectors, from basic vector design through clinical development. Dr Crystal has approved 14 applications of new investigational drugs in gene therapy and has published over 300 articles on gene therapy, with experience in CNS, heart, lung and liver disease.
“I am delighted to be working with LEXEO Therapeutics to move our broad academic portfolio into clinical development and ultimately bring it to patients,” said Dr Crystal. “LEXEO’s AAV-mediated gene therapy programs have the potential for wide applicability across a range of therapeutic indications, and in a single company pipeline, present an opportunity for the natural evolution of gene therapy from rare genetic diseases to more common diseases. “
The President of LEXEO Therapeutics, Dr Steven Altschuler, is currently the Managing Director of Ziff Capital Investments and was previously President of gene therapy biotechnology pioneer Spark Therapeutics, who was responsible for the first FDA-approved gene therapy, Luxturna®. , and was acquired by Roche in 2019 for $ 4.3 billion.
“LEXEO’s impressive leadership team, with Nolan’s years of experience in rare disease leadership at Pfizer, along with its pioneering scientific founder and syndicate of high-quality investors, will propel the development of the program pipeline.” promising and innovative society, ”said Dr Altschuler. “I am honored to have the opportunity to partner with Nolan and his team to create a premier gene therapy business. “
LEXEO Therapeutics has also appointed Dr. Jay Barth Executive Vice President and Chief Medical Officer to oversee Medical Affairs, Regulatory Affairs and Clinical Development. Dr Barth was previously Medical Director at Amicus Therapeutics and Senior Vice President, Clinical Development at PTC Therapeutics, where he oversaw all clinical development programs and Galafold approval.® for Fabry disease in Amicus, as well as Translarna®, the first treatment approved for Duchenne muscular dystrophy at PTC Therapeutics.
Other members of the LEXEO Therapeutics board include CEO R. Nolan Townsend, Sandip Agarwala of Longitude Capital, Bernard Davitian of Omega Funds and Mette Kirstine Agger of Lundbeckfonden Ventures.
Friedreich’s ataxia (FA) is a rare degenerative multisystem disease that affects approximately 1 in 50,000 people in the United States. FA is caused by a genetic mutation that disrupts the normal production of the protein frataxin, which is essential for the functioning of mitochondria (the energy-producing factories) in a cell. FA is inherited autosomally recessively, usually begins in childhood, and results in impaired muscle coordination (ataxia) that worsens over time, usually progressing to serious heart problems including hypertrophic cardiomyopathy and arrhythmias . FA is also associated with visual impairment, hearing loss, scoliosis, diabetes and speech disorders. Friedreich’s ataxia can shorten life expectancy, with heart failure being the most common cause of death. Supported by soon to be published de novo preclinical research, LX2006 is an intravenously administered AAV-mediated frataxine gene therapy treatment focused on the heart pathology of FA. The company is completing preclinical studies enabling IND and plans to launch a Phase 1 trial in 2021.
CLN2 disease (late childhood Batten disease) is an autosomal recessive lysosomal overload disease with less than 1000 cases worldwide, with a typical onset in children between 2 and 4 years of age. The disease is caused by mutations in the CLN2 gene, leading to progressive cognitive impairment, visual impairment, seizures and deterioration of motor development. LX1004 is an AAV-mediated gene therapy treatment that delivers CLN2 to the central nervous system. In December 2020, clinical data published in Science Translational Medicine found that a single administration of AAV-mediated CLN2 gene therapy (LX1004) slows the progression of CLN2 disease in children. Treatment with LX1004 was well tolerated, with minimal serious adverse events during the acute / postoperative period (0-14 days) and during the 18 month study period (14 days – 18 months). Once this Phase 1/2 study is complete, the company plans to advance the program to a pivotal study in 2022.
Alzheimer’s disease is the leading cause of late-onset dementia, characterized by progressive memory loss and cognitive decline in humans. APOE is an important cholesterol transporter and is in part linked to the pathogenesis of Alzheimer’s disease due to the development of amyloid plaques and tau tangles in the brain. People who inherit the APOE4 alleles are at a significantly higher risk of developing Alzheimer’s disease and at an earlier age of onset than people who inherit the APOE3 or APOE2 alleles, who are at normal and reduced risk, respectively. onset of the disease.ii LX1001 is an AAV-mediated gene therapy treatment that delivers APOE2 to the central nervous system of people with two APOE4 alleles (homozygous), via a CNS route of administration. A phase 1 clinical study is underway.
About LEXEO Therapeutics, Inc.
LEXEO Therapeutics is a fully integrated, New York-based biotechnology company currently based in the Alexandria Center.® for Life Science which aims to apply the transformational science of gene therapy to fight some of the world’s most devastating genetic and acquired diseases. LEXEO Therapeutics’ pipeline includes adeno-associated virus (AAV) mediated therapies primarily developed in the Department of Genetic Medicine at Weill Cornell Medicine. Beyond LEXEO Therapeutics’ flagship programs – which focus on rare and non-rare monogenic diseases (single gene mutation) – the company’s preclinical pipeline covers monogenic diseases, as well as inherited and acquired diseases in a wide range of patient population sizes and a range of unmet medical needs. Importantly, LEXEO Therapeutics will focus on advancing clinical programs through commercialization, with the goal of maintaining an ongoing research collaboration with the Department of Genetic Medicine at Weill Cornell Medicine to help advance the pipeline. preclinical of the company. For more information, please visit www.lexeotx.com Where LinkedIn.
LEXEO Therapeutics, Inc.
Sheryl Seapy, Group W2O