LogicBio Therapeutics’ Nasdaq IPO is slated for next week and the gene-editing player has set its terms—it plans to raise $75 million to advance its lead asset, a treatment for the genetic liver disease methylmalonic acidemia (MMA), into the clinic.
The Cambridge, Massachusetts-based biotech could raise up to $81 million if it hits the high end of its price range, according to a Renaissance Capital report. The IPO haul would fund a phase 1/2 trial for the LB-001, LogicBio’s gene-editing treatment for MMA, “through the initial data read-out, clinical development outsourcing, drug manufacturing and internal personnel costs,” the company said in its S-1, filed in September.
MMA is a disorder in which the body cannot process certain proteins and fats properly. It can be caused by mutations in several genes, though the NIH estimates that mutations in the MUT gene account for 60% of cases. Children develop the condition in early infancy and can experience symptoms such as weak muscle tone, developmental delay, enlarged liver and lethargy. MMA can lead to long-term complications, including chronic kidney disease, pancreatitis and intellectual disability.
Treatment focuses on carefully controlling the child’s diet—timing protein intake and using supplements. Liver and/or kidney transplants have been shown to help some patients break down methylmalonic acid. There is no cure.
LogicBio’s LB-001 aims to incorporate a functional copy of the MUT gene into the genome of MMA patients. The goal is to make the edit early in a patient’s life to head off severe disabilities. The treatment is based on the company’s GeneRide platform, a technology that uses homologous recombination that is designed to allow site-specific transfer of therapeutic genetic material without the use of promoters or nucleases. LogicBio says it also has access to a library of synthetic, non-pathogenic, recombinant adeno-associated viral (rAAV) vectors developed at Stanford that allows for better predictability of vector performance in clinical trials.
The IPO will also bankroll the discovery and preclinical development of a handful of candidates developed for liver indications. These include hemophilia B, Alpha-1 antitrypsin deficiency (A1ATD), which can cause lung and liver disease, and Crigler-Najjar syndrome, in which patients cannot clear bilirubin from the body.
The deal comes a year after LogicBio picked up a $45 million B round from the likes of Arix Bioscience, OrbiMed and Rothschild.
“Early intervention for rare genetic disorders in children is important and LogicBio is uniquely positioned at the forefront of this research area with its proprietary genetic therapy technology to deliver a durable cure for young patients with life-threatening genetic diseases and otherwise limited options. LogicBio has huge potential and, alongside its excellent team and investors, we look forward to supporting the company to achieve continued success in this area,” said Joe Anderson, Arix Bioscience CEO, at the time.