LogicBio Therapeutics Announces $50 Million in Financing to Develop Novel Gene Medicines for Pediatric Patients

Press release - 6/29/2017

In short
  • Combines two unique technology platforms to deliver a one-time treatment in pediatric diseases, while avoiding unwanted side effects
  • Focusing first on infants with inborn errors of metabolism, where there are currently few, if any, treatment options

LogicBio Therapeutics, Inc., a gene-therapy company with a mission to develop cures for early-onset life-threatening diseases, emerged from stealth mode to announce that it has successfully raised approximately $50 million in financing to date.

The investments were led by Arix Biosciences and OrbiMed. Edmond de Rothschild Investment Partners, Pontifax, and SBI Japan-Israel Innovation Fund also participated. LogicBio also announced a relocation from California to LabCentral in Kendall Square, Cambridge, Massachusetts. The funds and move will enable the startup to continue development of its GeneRide™ technology, a first-in-class, proprietary genome-editing platform, and to accelerate its lead programs to the clinic.

“We’ve been able to demonstrate the breadth of our technology in proof-of-concept studies across multiple preclinical models,” said LogicBio President and CEO Frederic Chereau. “Conducted in collaboration with toptier academic labs, these studies show that the GeneRide technology can provide stable, therapeutic levels of life-saving proteins after a single injection. We are passionate about taking the next step to deliver on the promise of this breakthrough technology for patients with early-onset rare diseases. In particular, we are focused on treating infants, which is a challenge for many gene therapies. And we’re excited to have such strong support from our investors to help us advance toward our goal of human proof-of-concept trials.”

A major challenge in applying traditional gene-therapy techniques in the pediatric population is the proliferation of cells in the growing tissue of a child, which dilutes the therapeutic benefit. In many rare genetic diseases, such as inborn errors of metabolism, symptoms arise in the first year of life and progress rapidly, often leading to death, so early intervention is critical. LogicBio is uniquely positioned to address this challenge. The company’s GeneRide technology hitches a therapeutic transgene to a native promoter without disrupting its normal function in the host genome, enabling permanent expression of a functional version of a missing or faulty gene. Thus − with just a single treatment early in a child’s life – LogicBio can potentially deliver a life-long cure from a devastating disease.

LogicBio’s co-founders Mark Kay, M.D., Ph.D., Adi Barzel, Ph.D., and Leszek Lisowski, PhD., M.B.A., developed the company’s two core technologies in the Kay Lab at Stanford University: 1) The GeneRide technology harnesses the natural power of homologous recombination to enable precise, site-specific transfer of therapeutic genetic material without the use of promoters or nucleases. This strategy minimizes the risk of carcinogenicity spurred on by strong promoters used in traditional gene-therapy approaches. 2) A library of novel synthetic, nonpathogenic recombinant adeno-associated viral (rAAV) vectors, designed specifically to enable significant clinical performance advantage over established clinical capsids.

The combination of LogicBio’s GeneRide technology and AAV vectors has the potential to maximize safety and efficacy in the practice of gene therapy,” commented Mark Kay, who is also chief science advisor to the company. A leading scientist in the fields of gene therapy and viral vector biology, Dr. Kay is an original founding member of the American Society of Gene and Cell Therapy, serving as its president from 2005 to 2006. In 2013, he was recipient of the society’s Outstanding Investigator Award.

Genetically defined congenital diseases can be profoundly devastating for both patients and families, yet for many of these conditions we have few treatment options, if any at all. LogicBio’s novel approach to treating these pediatric patients is the product of the perspicacious scientific founders and management team. LogicBio plans to develop products that will be disease modifying, rather than merely supportive, and we are pleased to be able to support a company that aims to produce transformational treatments for these patients,” said Daniel O’Connell, M.D., Ph.D., investment manager at Arix Bioscience.

We’re pleased with LogicBio’s progress since seeding the company in early 2016,” said Erez Chimovits, senior managing director at OrbiMed Israel. “We believe that the well-differentiated platform technology addresses some significant shortcoming of episomal gene-therapy approaches. We welcome the new investors and look forward to advancing breakthrough therapies to the clinic.”

Fred Chereau, Erez Chimovits, Mark Kay, and Daniel O’Connell have all been appointed to the LogicBio board of directors. They will be joined by Leon Chen, Ph.D., venture partner at Orbimed; Sofia Ioannidou, Ph.D., investment director at Edmond de Rothschild Investment Partners; and Tomer Kariv, J.D., founder and CEO of Pontifax.

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