Another gene therapy company is on its way to the public markets. But this one, LogicBio Therapeutics, is part of a newer crop of gene therapy developers that aim to overcome the potential shortcomings of the treatments on the market or in human trials. LogicBio’s technology is still preclinical, but the company aims to start human testing next year, and it is now planning an IPO to finance that research.
Cambridge, MA-based LogicBio set a preliminary $86 million target for its IPO. The company has applied for a listing on the Nasdaq exchange under the stock symbol “LOGC.” LogicBio aims to use the IPO cash to start its first clinical trial, for a rare inherited liver disease called methylmalonic acidemia (MMA).
Gene therapy has finally come of age after years of ups and downs. By introducing a functioning version of a faulty or missing gene into a cell. these treatments are intended to provide a long-lasting, if not permanent treatment for a genetic disorder. One product, for a genetic form of blindness, launched in the U.S. at the beginning of the year. More, for hemophilia, spinal muscular atrophy, and more, could follow, and several gene therapy developers are publicly traded. But even with the promise these treatments have shown, it’s unclear how long a therapy will last and who will respond. And when given to children, it’s possible some gene therapies might lose their effect over time as they grow and their cells divide.
LogicBio is one of several emerging companies trying to address these gene therapy issues. Its technology, called GeneRide, was developed to insert genes into specific locations in the genome. The company says this approach enables the transferred genetic material to remain stable as cells divide and tissue grows. The LogicBio gene therapy is inserted into the genome via a naturally occurring DNA repair process called homologous recombination. This process avoids using enzymes that cut DNA—a feature of gene-editing technologies such as CRISPR. These enzymes can cause unwanted and potentially dangerous modifications to the DNA, which in turn could cause tumors to form, LogicBio says in its prospectus. (LogicBio isn’t alone in trying to harness homologous recombination; Homology Medicines (NASDAQ: FIXX), which went public in March, is in the mix as well.)
LogicBio is starting with a potential treatment for MMA. Infants who have the condition can’t process certain fats and proteins, which leads to vomiting, dehydration, weak muscles, and developmental delays, among other problems. MMA has no FDA-approved therapies. Treatment for the disorder includes a low-protein and high calorie diet, antibiotics, and organ transplant, according to the Genetic and Rare Diseases Information Center. Depending on the severity of the disorder, MMA can be fatal.
The company’s MMA gene therapy, LB-001, is meant to help MMA patients produce more of the protein that they lack. LogicBio plans to start a Phase 1/2 clinical trial by the end of next year.
LogicBio is also developing potential treatments for hemophilia B; alpha-1 antitrypsin deficiency, which can lead to lung disease; and the rare disease Crigler Najjar Syndrome, which renders the body unable to clear a compound produced in the breakdown of old red blood cells.
LogicBio is led by CEO Fred Chereau, an industry veteran who has held senior roles at aTyr Pharma, Shire (NASDAQ: SHPG), and Genzyme. The company has raised $50 million in venture financing, most recently a $45 million Series B round of financing last year. LogicBio’s largest shareholder is OrbiMed, which holds a 35.3 percent stake in the company, according to the filing. Other large shareholders include Arix BioScience (LSE: ARIX), Pontifax, and BioDiscovery 5.
The company was founded in 2015, based on technologies licensed from Stanford University and the University of Texas System. The company spent $3.6 million on R&D in the first half of 2018, and had $18.2 million in cash at the end of June.
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