Several leading companies in the gene editing space participated in a panel session at MOTM in Carlsbad, CA, noting that base editing has already entered the clinic and the first in vivo CRISPR approaches are progressing in clinical trials. However, these companies foresee future advances and innovation that will help take genome editing to the next level.

Devyn Smith, PhD, CEO of Arbor Biotechnologies, told the panel that the promise of gene editing is garnering so much interest because the technology can "edit and completely fix a problem in the DNA" resulting in the "ability not to treat disease but to actually cure it."

At the same time, despite advancements in curative gene editing-based therapies, Smith acknowledged that limitations with current approaches remain a hurdle to leveraging this modality to its full potential. As an early-stage life sciences company, Arbor Biotechnologies is working to uncover unique CRISPR enzymes that can power a wide range of applications, including therapeutics and manufacturing, and contend they are using gene editing technologies across cell and gene therapy in ways existing tools cannot.

"We have made tremendous progress but there's still a lot to do," said Fred Chereau, CEO of clinical-stage genetic medicine company LogicBio Therapeutics. "Gene editing is the new shiny thing but it's still very early...I think we have to be very humble with the science and continue to work step by step and continue to share the learnings."

LogicBio's GeneRide proprietary platform for genome editing uses a synthetic viral vector to deliver the corrective transgene to the nuclei of patients' cells through an infusion, which the company contends has the potential to provide safety and efficacy benefits over existing technologies.

Mark Shearman, PhD, chief scientific officer at Editas Medicine, a clinical-stage biotech company developing therapies for rare diseases based on CRISPR gene editing, made the case during the panel that "with any new technology, obviously the focus is on clinical data and proving that it's safe and efficacious."

Last week, Editas announced ex vivo and in vivo preclinical data supporting its experimental medicine EDIT-103 for the treatment of rhodopsin-associated autosomal dominant retinitis pigmentosa (RHO-adRP). EDIT-103 is a mutation-independent CRISPR-Cas9-based, dual adeno-associated virus (AAV) vectors "knockout and replace" therapy to treat RHO-adRP, a hereditary progressive form of retinal degeneration.

Albert Seymour, PhD, CEO of Homology Medicines, pointed out to the panel that many companies in the gene editing space are currently targeting rare monogenic diseases -- caused by a defect in a single gene -- because the biology is relatively straightforward.

"Where gene editing is right now, to the truest definition, is going in and modifying DNA at one site. That's why you see it in monogenic disorders," Seymour said. "You have to know exactly what part of the genome is contributing to the disease you're trying to treat."

Homology Medicines' clinical programs include HMI-103, a gene editing candidate for phenylketonuria (PKU); HMI-203, an investigational gene therapy for Hunter syndrome; and HMI-102, an investigational gene therapy for adults with PKU. The company's proprietary platform is designed to utilize a family of 15 human hematopoietic stem cell-derived adeno-associated virus (AAVHSCs) vectors to deliver genetic medicines in vivo through a gene therapy or nuclease-free gene editing modality.

According to Seymour, one of the biggest current technology gaps in the industry is in delivery to the right cell. Seymour noted that while "we can get to certain organs well...there are areas of the body that we just can't get to currently with some of the technologies."

Nonetheless, Shearman warned that "it's easy to get carried away sometimes with what you could do" and when it comes to delivery and manufacturing it's important ultimately to make the editing technologies as simple as possible.

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