Precision BioSciences (NasdaqCM:DTIL) Conference Transcript

Summary of Precision BioSciences Conference Call Company Overview - Company: Precision BioSciences (NasdaqCM:DTIL) - Industry: In-vivo gene editing - Location: Durham, North Carolina - Focus: Developing gene editing therapies for chronic hepatitis B and Duchenne muscular dystrophy (DMD) [2][3][4] Key Programs PBGene-HBV (Hepatitis B) - Target Population: 300 million people globally with chronic hepatitis B [2] - Current Status: Program initiated in the clinic late 2024, with ongoing data generation throughout 2025 and into 2026 [3][4] - Mechanism: Aims to eliminate cccDNA, the root cause of hepatitis B, rather than just reducing S antigen levels [9][10][12] - Clinical Trial: Phase 1 trial named "Eliminate B" with three cohorts, focusing on safety and efficacy [13][14] - Safety Profile: Well-tolerated across all doses, with manageable side effects [15][20] - Data Trends: Early cohorts show promising reductions in S antigen levels, with cohort three demonstrating sustained reductions [16][18] - Future Plans: Completion of cohort three dosing, potential to stop nucleoside analogs, and test for a cure [20][21] PBGene-DMD (Duchenne Muscular Dystrophy) - Target Population: Affects boys with a genetic disease leading to muscle loss and early mortality [21][22] - Current Status: IND filing expected by the end of 2025, with clinical trials anticipated to start in early 2026 [25][26] - Mechanism: Gene editing approach targeting mutations in exons 45 to 55, potentially benefiting up to 60% of DMD patients [22][24] - Clinical Trial Design: Phase 1, 2, 3 study design with a focus on safety and efficacy [25][26] Financials - Recent Financing: Raised $75 million from existing and new investors, providing a cash runway through 2028 [4] - Investment Strategy: Focused on two key programs to maximize capital efficiency and create value inflection points [35] Market Context - Unmet Needs: Both hepatitis B and DMD represent significant unmet medical needs, with current treatments lacking effectiveness [21][22][27] - Competitive Landscape: Current standard of care for hepatitis B involves nucleoside analogs, which do not address cccDNA, resulting in low functional cure rates (1%-3%) [29][30][31] Strategic Partnerships - PBGene-HBV: Potential for strategic partnerships for commercialization, especially for larger phase three trials [32] - PBGene-DMD: Opportunities for partnerships exist, but the company is capable of advancing this program independently [33] Future Outlook - Data Readouts: Anticipated data readouts for both programs in 2026, with potential for a Biologics License Application (BLA) for DMD by the end of 2028 [26][27] - Investor Engagement: Emphasis on demonstrating the effectiveness of gene editing to attract further investment and partnerships [35]