LogicBio Therapeutics Launches Development Program in Methylmalonic Acidemia (MMA)
Published: May 09, 2018
“We’re driven to help pediatric patients avoid the debilitating consequences of MMA by giving physicians safe, durable options for early intervention,” said LogicBio Chief Executive Officer Fred Chereau. “We believe that LB-001, our lead clinical candidate, has strong potential to revolutionize the treatment of this devastating disease, and we are moving rapidly towards clinical development.”
Primarily caused by mutations in the MUT gene, MMA is a rare, autosomal recessive disease that prevents the body from properly processing certain fats and proteins, resulting in a toxic accumulation of metabolites that can cause life threatening decompensations in infants and children. This buildup can lead to significant morbidity and mortality, including infections, neurodevelopmental disabilities and chronic kidney disease. There is no therapy available to treat severe MMA; physicians can only manage symptoms with a combination of a strict low-protein/high-calorie diet, vitamin B injections, antibiotics and, in some cases, liver and kidney transplantation.
“MMA takes an enormous toll on patients and their families and we have no medications to help them,” said Jerry Vockley, M.D., Ph.D., professor of Pediatrics and Human Genetics at the University of Pittsburgh, and director of the Center for Rare Disease Therapy at Children’s Hospital of Pittsburgh where he treats children with MMA. “Using genome editing to give pediatric patients a functioning version of the gene they lack would be a huge advance. We are encouraged by the potential for a gene therapy like LB-001 to vastly improve the lives of these patients.”
LB-001 is an investigational, first-in-class pediatric genome editing therapy based on LogicBio’s GeneRide™ technology. GeneRide™ enables site-specific integration and lifelong expression of therapeutic transgenes, without the use of exogenous promoters or nucleases. LB-001 is designed to incorporate a functioning version of the faulty MUT gene into the genome of MMA patients.
In preclinical studies conducted at the National Institutes of Health in the lab of Charles P. Venditti, M.D., Ph.D., senior investigator, and head of the Organic Acid Research Section in the National Human Genome Research Institute (NHGRI), an AAV GeneRide™ vector was used to rescue neonatal lethality seen in MMA mice. Hepatic correction was durable for over one year, and suggestive of a selective growth advantage. The NHGRI team is expected to present additional preclinical data at next week’s American Society of Gene & Cell Therapy (ASGCT) conference.
As part of the LB-001 program, LogicBio will also initiate a natural history study of MMA in the next year. The MMA study will provide critical background knowledge of the disease as LB-001 advances toward an IND filing and clinical trial in 2019.
“The burden of MMA on families is significant,” said Kathy Stagni, executive director of the Organic Acidemia Association, a nonprofit advocacy group for families living with MMA and other organic acidemia metabolic disorders. “We are encouraged by LogicBio’s early intervention approach and look forward to collaborating with the company in its effort to develop an innovative genetic therapy for patients with this progressive and often fatal disease.”
About LogicBio Therapeutics
LogicBio Therapeutic is a genetic medicine company, founded to deliver the benefits of genome editing to the fight against early onset childhood diseases. Launched in 2016 by pioneers in gene therapy from Stanford University and funded by leading life science investors, LogicBio is developing a pipeline of safe and durable treatments for pediatric indications. Our core technology, GeneRide™, enables the site-specific integration of a therapeutic transgene, in a nuclease-free and promotor-less approach by relying on the native process of homologous recombination to drive lifelong expression. With a world-class global team headquartered in Cambridge, Mass., LogicBio is committed to developing medicines that will transform the lives of pediatric patients and their families.
For more information, please visit www.logicbio.com.
Ten Bridge Communications
Paul Goldsmith, 617-697-3479
Source: LogicBio Therapeutics, Inc.