Following last year’s stellar initial public offering in October, where it raised $80.5 million, about $10 million over initial expectations, LogicBio Therapeutics is planning a gene-editing clinical trial in children.
Based in Cambridge, Mass., LogicBio focuses on gene therapy and gene editing. Its technology came out of Stanford University and led to its GeneRide tech platform. This platform is designed to integrate corrective genes into a patient’s genome in a precise and stable way, using a natural DNA repair process called homologous recombination.
The company plans to submit an application to the U.S. Food and Drug Administration (FDA) to launch its first clinical trial by the end of this year. The trial would be to treat methylmalonic acidemia, a hereditary disease that leaves patients unable to metabolize certain proteins and fats. It usually affects infants and children, and results in a range of symptoms, including muscle weakness and intellectual disabilities.
Children with the disease who make it past infancy have a life expectancy of 20 to 30 years. It affects about one in 50,000 children.
Instead of using CRISPR for gene editing, LogicBio’s GeneRide platform uses two homologous gene strands to transport a corrected gene into the DNA sequence. CRISPR has some yet-unresolved problems regarding off-target gene edits that the GeneRide system does not seem to have.
The Boston Business Journal notes, “Homologous recombination has historically had a low rate of success compared to other gene therapies,” according to Kyle Chiang, vice president of product strategy. “LogicBio’s challenge is two-fold, because gene therapies are often administered in the liver, but infants and children’s liver cells divide and multiply much more frequently than in adults, degrading the treatment.”
But LogicBio’s approach utilizes the albumin gene, which is very active in the liver, resulting in better integration. The company also believes its approach could be used in muscles and other body sites.
The company’s chief executive officer Frederic Chereau also indicated it is looking for a commercial partner with a background in muscular diseases and disorders.
The company currently has 32 staffers and plans to hire almost 20 more this year.
In November 2018, the company inked a partnership deal with Children’s Medical Research Institute (CMRI) of Australia to develop new viral vectors. They formed a new AAV Development Program to develop next-generation synthetic adeno-associated virus (AAV) capsids. LogicBio holds exclusive worldwide commercial rights to vectors that come out of the partnership, with a goal of commercializing them as widely as possible.
The AAV Development Program is led by Ian Alexander, Head of CMRI’s Gene Therapy Research Unit and Leszek Lisowski, leader of CMRI’s Translational Vectorology Group. Lisowski is a scientific co-founder of LogicBio, who worked with Mark A. Kay at Stanford University.
“We are confident that we can further improve on the performance of current AAV vectors,” Lisowski stated at the time, “expanding their utility in a range of tissues, while also improving manufacturability and reducing cost.”
Most gene therapies utilize adeno-associated viruses, but for the most part, they haven’t changed in the last 10 years. LogicBio developed a synthetic liver-tropic AAV capsid, AAV-LK03, which is being evaluated by the company to deliver gene therapies using GeneRide.