Clinical Trials

Date: 2018-05-08

Type of information: Initiation of development program

phase: preclinical

Announcement: initiation of development program

Company: LogicBio Therapeutics (USA - MA)

Product: LB-001

Action mechanism:

• gene editing. 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.

Disease: methylmalonic acidemia (MMA)

Therapeutic area: Rare diseases - Genetic diseases - Metabolic diseases

Country:

Trial details:

Latest news:

• • On May 8, 2018, LogicBio Therapeutics,announced it has initiated a development program targeting methylmalonic acidemia (MMA)—a rare, life-threatening pediatric metabolic disease.
• 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.
• 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 the 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.

 

Is general: Yes