LogicBio Raises $42 Million to Support Gene Editing Platform

Rare Daily Staff

LogicBio Therapeutics said it raised $42 million through a public offering of 7 million shares of its common stock at $6 per share.

In addition, LogicBio granted underwriters a 30-day option to purchase up to an additional 1.1 million shares at the public offering price less underwriting discounts and commissions.

LogicBio intends to use the funding to support clinical development of LB-001, in phase 1/2 studies as a treatment for methylmalonic acidemia (MMA), a rare life-threatening metabolic disorder, and to advance the development of its targeted delivery platforms GeneRide and Next Generation Capsid, among other things.

Methylmalonic acidemia (MMA) is an inherited condition in which the body is unable to process certain proteins and fats properly. Signs and symptoms usually appear in early infancy and vary from mild to life-threatening. Affected infants can experience vomiting, dehydration, weak muscle tone, developmental delay, lethargy, hepatomegaly, and failure to thrive. Long-term complications can include feeding problems, intellectual disability, chronic kidney disease, and pancreatitis. Without treatment, this condition can lead to coma and death in some cases. Mutations in the MUT, MMAA, MMAB, MMADHC, and MCEE genes cause methylmalonic acidemia. It is inherited in an autosomal recessive fashion.

LogicBio’s proprietary genome editing technology platform, GeneRide, enables the site-specific integration of a therapeutic transgene without nucleases or exogenous promoters by harnessing the native process of homologous recombination. The company is also developing a Next Generation Capsid platform for use in gene editing and gene therapies.

Recently, the company partnered with Takeda to advance LB-301, an experimental therapy using LogicBio’s proprietary genome editing technology for the treatment of Crigler-Najjar syndrome, a rare monogenic pediatric disease caused by a deficiency in the liver-specific UGT1A1 gene resulting in severely high levels of unconjugated bilirubin in the blood starting at birth, with lifelong risk of permanent neurological damage and death.

Under the agreement, Takeda will provide funding for the research program under the collaboration agreement and will have an exclusive option to negotiate an exclusive, worldwide license to LogicBio’s LB-301 program.