Editas Medicine, Inc. (NASDAQ:EDIT) Q4 2019 Earnings Conference Call - Final Transcript
Feb 26, 2020 • 05:00 pm ET
Cynthia L. Collins
We accelerated our efforts to develop engineered allogeneic NK cell medicines for cancer, through newly formed research collaborations. We enabled our development of healthy donor NK cell medicines for solid tumors with cell expansion technology from Sandhill Therapeutics, and we are advancing iPSC-derived NK cell medicines using technology from BlueRock Therapeutics. And we added outstanding executive leadership with the appointments of Judith Abrams as our Chief Medical Officer; Michelle Robertson as our Chief Financial Officer, and Harry Gil as our Senior Vice President of Operations. I am pleased to have Judith and Michelle joining me on the call today.
These accomplishments give us momentum into the coming year. In 2020, we plan to announce dosing of patients in the first quarter in the Brilliance Phase 1/2 trial of EDIT-101 in collaboration with our partner, Allergan. We plan to nominate a development candidate for autosomal dominant retinitis pigmentosa 4 or RP4, file an IND for EDIT-301 for the treatment of sickle cell disease, initiate IND-enabling studies for an allogeneic healthy donor NK cell medicine candidate to treat solid tumors, and finally, present in vivo preclinical proof of concept data for an engineered iPSC-derived NK cell medicine to treat solid tumors.
Now, let me turn the call over to our Chief Medical Officer, Judith Abrams, to update you on our Phase 1/2 clinical trial of EDIT-101.
Judith R. Abrams
Thanks, Cindy. It's my pleasure to join all of you on the call today. As Cindy mentioned, last year we opened the Brilliance Phase 1/2 study for patients with LCA10 for enrollment. Enrollment activity has accelerated in recent months and we expect an announcement on the first patient dosing in the first quarter of this year. As a reminder, the first patient dose in the Brilliance clinical trial will mark a significant milestone towards delivering on the promise and potential of CRISPR medicine to durably treat devastating diseases such as LCA10.
I'm also pleased to share that as our research pipeline continues to progress to the clinic, we are building out our senior leadership in our clinical organization.
I'll now turn over the call to our Chief Scientific Officer, Charlie Albright, to discuss our broader pipeline.
Thank you, Judith, and thank you all for joining us on the call. Following EDIT-101, our next in vivo ocular program is EDIT-102 for the treatment of Usher Syndrome 2A or USH2A. Like LCA10, USH2A is an inherited retinal disease that affects photoreceptors and leads to blindness. At the product level, EDIT-102 is nearly identical to EDIT-101, in that EDIT-102 uses the same AAV5 delivery vector, proprietary Staph aureus Cas9 enzyme and photoreceptor-specific promoter as does EDIT-101. Preclinical study support the advancement of EDIT-102 into an IND-enabling study, since we have demonstrated editing levels, mRNA transcriptional levels and phenotypic restoration that are consistent with therapeutic benefit. Based on these data, we delivered a data package for EDIT-102 to Allergan for potential licensing and development as part of our strategic alliance we formed in 2017.
Our learnings with EDIT-101 and