William Blair initiated research coverage of Intellia Therapeutics, Inc. (NTLA $95.86), a clinical-stage genome-editing company focused on using CRISPR-Cas9 technology to create in vivo and ex vivo therapies for diseases with high unmet need.

Analyst Raj Prasad expects several updates from the company in 2022, including data from the Phase I study of lead candidate NTLA-2001 in ATTR (transthyretin amyloidosis) patients with polyneuropathy (ATTRv-PN); completion of enrollment in the NTLA-2001 Phase I study in ATTRv-PN and ATTR patients with cardiomyopathy (ATTR-CM, a large TAM expansion) arms; interim data from its Phase I/II trial with NTLA-2002 in hereditary angioedema; two new in vivo development candidates; enrollment of patients in a Phase I/IIa study of its lead ex vivo program, NTLA-5001, in acute myeloid leukemia; and the nomination of a wholly owned allogeneic ex vivo candidate.

“Intellia has established itself as a pioneer in the in vivo application of CRISPR-Cas9 editing,” Dr. Prasad said, “and has leveraged its platform technology in combination with additional proprietary capabilities, such as nonviral delivery, to address unmet need across genetic diseases, immuno-oncology, and autoimmune indications. The company has two partnerships with large biotechnology companies: an in vivo partnership with Regeneron that includes up to 15 targets with a mix of co-developed and licensed programs and an ex vivo partnership with Novartis that is focused primarily on a sickle cell disease program called OTQ923, along with other CAR-T, hematopoietic stem cell, and ocular stem cell indications that are yet to be disclosed.”

Dr. Prasad continued, “For NTLA-2001, we see a $1 billion-plus market opportunity in the ATTR-PN population that expands to a multibillion opportunity if successful in the larger ATTR-CM population. We assume a 2026 U.S. launch for NTLA-2001 in the ATTR-PN subpopulation and a 2027 launch in the ATTR-CM population. While we expect proof-of-concept clinical data from NTLA-2002 in the second half of the year, we believe the asset is de-risked from the Phase I NTLA-2001 data because of similarities in product composition. From the earlier-stage pipeline, both NTLA-5001 in AML and NTLA-3001 in A1ATD are using approaches that would expand the platform potential of Intellia’s CRISPR-Cas9 technology.”

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