Editas Medicine, Inc. Q2 FY2024 earnings call

• Reni-cel: Shared RUBY and EdiTHAL clinical data at EHA 2024; RUBY enrollment strong with adult and adolescent cohorts progressing; manufacturing process for reni-cel is robust with low failure rate. • In-vivo pipeline: Focus on functional upregulation strategy, targeting rare orphan diseases, lead discovery in hematopoietic stem cells and other tissues; leveraging indel technology for in-vivo therapeutics. • Financials: Cash, cash equivalents, and marketable securities as of June 30, 2024, were $318 million, down from $377 million on March 31, 2024; burn rate higher due to increased external R&D expenses related to reni-cel program.

No specific financial performance by product segments with absolute terms and revenue contribution % provided in the transcript.

• Expect to share additional clinical data for RUBY and EdiTHAL trials by end of 2024. • On-track to establish in-vivo pre-clinical proof-of-concept for an undisclosed indication by end of 2024. • Continued discussions with FDA regarding BLA package similar in scope to gene editing medicines field.

• Forward-looking statements may differ materially from actual results due to factors in the Risk Factors section of the most recent Annual Report on Form 10-K and subsequent filings. • Uncertainties in clinical trial outcomes, regulatory approvals, and market adoption of gene editing therapies.

Q: Ask about the pre-clinical proof-of-concept from the in-vivo program, including details on data, model type, and off-target editing profile.

A: Linda Burkly stated they are on-track to establish in-vivo pre-clinical POC for an undisclosed indication by end of year, evaluating biodistribution, editing efficiency, target modulation, and tolerability; details on species and timing of data disclosure to be shared later.

Q: Regarding LNP in strategy and proof-of-concept readout indication optimization.

A: Linda Burkly mentioned evaluating LNPs with partners for different tissue cell types, but specifics on targeting and optimization to be shared later when disclosing data.

Q: Timeline to BLA for reni-cel, considering adolescent cohort enrollment.

A: Baisong Mei noted RUBY has dosed over 20 patients, continuing to dose, and they consider CATXV approval as a benchmark, working towards BLA data package but final timeline alignment with FDA needed.

Q: Learnings from Kaskavi and Liginia launches for reni-cel launch.

A: Caren Deardorf mentioned fast follower timing as an advantage, anticipating shorter onboarding cycles for reni-cel launch based on Vertex and Bluebird examples.

Q: Average process time from patient enrollment to dose for adolescents vs adults, and clarification on indel technology upregulation.

A: Baisong Mei said enrollment to dosing varies, shorter for adolescents due to better health; Linda Burkly clarified indel technology is functional upregulation, not knockdown, disrupting repressor binding sites to upregulate gene expression like in reni-cel.

Q: IP licensing opportunities in future.

A: Erick Lucera stated they have strong IP from Harvard, MIT, and Broad, open to conversations with companies for bespoke licensing structures, with many ongoing programs and partnerships.

Q: Readiness towards BLA for reni-cel, including additional data needed.

A: Gilmore O’Neill mentioned ongoing discussions with FDA, confident in progress of clinical, preclinical, and manufacturing data, including off-target editing data which is robust using ASCAS12a.

Q: First-in-class or best-in-class approach for in-vivo assets.

A: Linda Burkly said differentiated functional upregulation allows targeting areas others can't with knockdown strategies, enabling first-in-class or best-in-class opportunities.

Q: In-vivo editing approach and number of diseases amenable to indel mediated functional upregulation.

A: Linda Burkly explained functional upregulation targets loss of function/deleterious mutations, many genetic diseases are amenable as they involve regulatory elements; Gilmore O’Neill added focus on non-coding genome regulatory elements.

Q: In-vivo delivery and target selection, and BEAM-101 data expectations.

A: Linda Burkly mentioned targeting hematopoietic stem cells and liver with LNPs, pragmatic approach based on delivery; Gilmore O’Neill said they look forward to BEAM-101 data but are confident in reni-cel's differentiated profile.

Q: Thoughts on HHS decision on fertility preservation for gene therapy patients with sickle cell disease, and partnering reni-cel.

A: Caren Deardorf was disappointed by the decision, hoping for reversal, and Erick Lucera stated they consider partnering outside the U.S. for optimal patient access and shareholder returns.

Q: Clinical differentiation for reni-cel and patient population size for in-vivo program.

A: Baisong Mei discussed monitoring end organ damage, cardiovascular, etc., with data expected at ASH; Gilmore O’Neill said initial in-vivo focus on rare orphan diseases with market size considerations for ROI.

Q: Follow-up on reni-cel BLA and adolescent cohort inclusion in label.

A: Baisong Mei said adolescent cohort enrollment completed, and they aim for broader age cohort inclusion in label with FDA alignment.

Q: In-vivo program target gene regulation and CAFC patent dispute timing.

A: Gilmore O’Neill explained both haploinsufficiency and recessive gene defect scenarios are possible; CAFC oral presentations in May, with decision anticipated before end of year.

Q: Fertility support in clinical trials and market impact if not provided post-approval.

A: Baisong Mei mentioned fertility support in trials; Caren Deardorf discussed CMMI model and commercial settings, expecting minimal market impact by reni-cel launch.

Q: Targeting Middle East region and launch strategy.

A: Caren Deardorf stated they continue to consider partnering outside the U.S. to target regions like the Middle East for reni-cel access.