Editas Medicine (EDIT -2.43%) CEO Katrine Bosley opened her comments at the J.P. Morgan Healthcare Conference on Wednesday by stating that it's "an incredibly exciting time" for Editas right now. Investors might not share that excitement after Editas' dismal stock performance in 2018. But they probably should share the optimism.
Bosley painted an encouraging picture for the next few years of progress for Editas. She was joined by other Editas executives in a question-and-answer session following her presentation that shed even more light on what's around the corner. Here are four key things discussed at the J.P. Morgan conference that point to a really bright future for Editas Medicine.
1. First in vivo CRISPR clinical trial starting soon
The most important thing by far for Editas in the immediate future is that the biotech will soon begin its first clinical study. And it will make history as the first in vivo (in the body) clinical trial in humans for a CRISPR gene editing therapy.
Editas and partner Allergan (AGN) plan to screen patients in the first half of 2019 for a phase 1/2 clinical study evaluating CRISPR therapy EDIT-101 in treating Leber congenital amaurosis type 10 (LCA10). LCA10 is the leading cause of inherited blindness. Dosing of patients will begin in the second half of the year.
The goal for EDIT-101 is to rescue vision in LCA10 patients by editing the genetic mutation that causes the eye disease. Bosley noted that the therapy achieved gene editing levels well above the predicted therapeutic threshold in both transgenic mice and nonhuman primates. She also pointed out data showing that EDIT-101 was well tolerated in nonhuman primates. This efficacy and safety profile in preclinical testing makes Editas hopeful that similar results will be achieved in human clinical studies.
2. Ability to "rinse and repeat"
LCA10 is just the beginning for Editas, though. Bosley expressed confidence that lessons learned in developing EDIT-101 for LCA10 will enable the biotech to repeat its formula for success in other genetic eye diseases.
In particular, she said that Editas Medicine's adeno-associated virus (AAV) method of delivering CRISPR therapies via a targeted local injection could be used in other programs. The data package for gaining a green light from the FDA to begin clinical studies for these other programs will also be similar to what was submitted for EDIT-101.
Next up on the list of indications that Editas will target with an in vivo CRISPR therapy is Usher syndrome type 2A (USH2A), another genetic eye disease. Editas Chief Scientific Officer Charlie Albright said that the company is conducting a final study "to nail down the candidate" for USH2A to advance to clinical testing. But Editas' approach will be very similar to that used to treat LCA10, which means the company's path to clinical studies should be a relatively smooth one.
There are other eye diseases that Editas and Allergan could target as well. And over the longer term, Albright said that Editas' AAV delivery approach could "open up gene editing more broadly" to other tissues.
3. A better approach for hematology cell therapies
While Editas is leading the way for in vivo CRISPR therapies, the company is also developing potentially transformative cell therapies. Bosley sounded especially excited about the biotech's prospects in treating rare blood disorders sickle cell disease (SCD) and beta-thalassemia using CRISPR to edit the genes in hematopoietic stem cells (HSC).
She stated confidently that Editas' approach to HSC gene editing -- called globin locus editing -- was better than the competing approach used by CRISPR Therapeutics. In particular, Bosley said that Editas' approach maintains the functionality of HSC cells more effectively than its rival's approach does.
Editas is behind CRISPR Therapeutics in moving into clinical studies. CRISPR Therapeutics and its partner Vertex Pharmaceuticals already have phase 1/2 studies in progress for CRISPR gene therapies targeting SCD and beta-thalassemia. But Albright said that Editas is "very close" to choosing a candidate to advance to clinical trials.
4. Strong intellectual property protection
Katrine Bosley also highlighted Editas Medicine's intellectual property (IP) for its therapies. She stated that Editas has "the broadest and deepest" IP in the CRISPR world.
In its early days as a company, Editas secured rights to CRISPR patents from the Broad Institute, Harvard, Massachusetts Institute of Technology (MIT), Duke University, and Massachusetts General Hospital. It also licensed IP for the CRISPR/Cpf1 gene editing approach discovered by Editas Medicine co-founder Feng Zhang.
Editas claims over 70 issued patents (including several covering EDIT-101) plus 600 patent applications pending approval. Bosley also noted that a September 2018 federal court decision strengthened Editas Medicine's IP foundation.
The future of medicine
Bosley's final comments of her presentation at the J.P. Morgan conference were perhaps the most significant. She said that "there are moments in time when science really astonishes us." Then she added that "this is actually one of those moments in time."
She went on to say that the use of CRISPR gene editing to treat diseases is "what the future of medicine looks like." Katrine Bosley is probably right. And if it's what the future of medicine looks like, the future for Editas Medicine should be really bright indeed.
Check out the latest Editas Medicine earnings call transcript.