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

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."

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.