DTIL News & Events

iECURE announced positive results from a preliminary analysis of the completed low-dose cohort of ECUR-506 from the ongoing OTC-HOPE clinical trial in neonatal-onset ornithine transcarbamylase, OTC, deficiency. Data were presented at the Society for Inherited Metabolic Disorders, SIMD, 2026 Annual Meeting. The first infant treated with ECUR-506 demonstrated a durable clinical response, with sustained discontinuation of standard-of-care therapies and no hyperammonemic events through 18 months post dose, as of February 11, 2026. In addition, participants treated with the low dose of ECUR-506 experienced statistically significant reductions in the annualized rates of HAEs and hyperammonemic crises, with 57% and 65% reductions, respectively, in pre-dose to post-dose rates of events per person-year, as of April 20, 2026.

Precision BioSciences announced new preclinical data from its Pbgene-DMD program in an oral presentation at the American Society of Gene & Cell Therapy, ASGCT, 2026 Annual Meeting in Boston, Massachusetts. The new data show that treatment with Pbgene-DMD in early-juvenile mice resulted in significantly higher efficacy across key skeletal and respiratory muscles than treatment in late-juvenile mice over a comparable timeframe. This new data further supports evaluating Pbgene-DMD in younger DMD patient populations, including the 2- to 3-year-old patients, who are a key demographic of the ongoing Phase 1/2 Function-DMD trial evaluating Pbgene-DMD in boys ages 2 to 7. Key data: New preclinical data show Pbgene-DMD drove up to a 3x higher dystrophin protein restoration in skeletal muscle and up to 12x higher in respiratory muscle in early-juvenile mice compared to late-juvenile mice; Findings further support evaluation of Pbgene-DMD in DMD patient populations as young as 2 years of age; Greater efficacy with earlier intervention further differentiates Pbgene-DMD from microdystrophin approaches

As of March 31, 2026, Precision had approximately $125.8 million in cash, cash equivalents, and restricted cash. The Company expects that existing cash and cash equivalents, continued fiscal and operating discipline, and availability of Precision's at-the-market facility will fund the Company's cash runway through 2028. Based on its expected cash runway, Precision believes it is sufficiently capitalized to achieve PBGENE-HBV and PBGENE-DMD data milestones through 2028.

Reports Q1 revenue $10.8M, consensus $4.05M. "During the first quarter, we continued to execute on our two clinical stage in vivo gene editing programs. We advanced PBGENE-HBV into new cohorts in the ELIMINATE-B trial and drove PBGENE-DMD through Investigational New Drug approval and activated our first clinical site as we prepare to dose the first patient in the FUNCTION-DMD trial," said Michael Amoroso, Chief Executive Officer of Precision BioSciences. "We remain focused on disciplined execution in 2026 as we generate additional clinical data from the ELIMINATE-B trial and enroll more sites and patients in the FUNCTION-DMD trial. We believe our ARCUS platform and targeted development strategy position us to deliver multiple important clinical and operational milestones this year."