Precision Biosciences Stock Price, News & Analysis

Precision BioSciences (NASDAQ: DTIL) announced initial results from the ELIMINATE-B Phase 1 trial of PBGENE-HBV, their novel gene editing therapy for chronic Hepatitis B. The first cohort of three patients received the lowest dose level (0.2 mg/kg) of PBGENE-HBV, which uses ARCUS-encoding mRNA in lipid nanoparticles.

Key findings include:

• The treatment was safe and well-tolerated with no Grade ≥2 treatment-related adverse events
• Two of three participants showed substantial reduction in Hepatitis B surface antigen (HBsAg)
• The trial is designed for three dose administrations per dose level

The study is currently enrolling HBeAg-negative chronic Hepatitis B patients in Moldova, Hong Kong, and New Zealand, with plans to expand to the U.S. and U.K. Precision plans to share detailed clinical data throughout 2025.

Precision BioSciences (NASDAQ: DTIL) announced key leadership changes to strengthen its in vivo gene editing programs. Cindy Atwell has been promoted to Chief Development and Business Officer, overseeing development functions including clinical, regulatory, and business development. She will supervise the PBGENE-HBV program and future in vivo initiatives.

Dr. Cassie Gorsuch has been elevated to Chief Scientific Officer, leading non-clinical development and gene therapy discovery. She will manage preclinical proof of concept and IND-enabling data for clinical studies advancement. The company's PBGENE-HBV program remains on schedule with Phase 1 clinical data expected in 2025.

Additionally, Dr. Sam Wadsworth has retired from his Director role on the company's Board.

Precision BioSciences (DTIL) announced positive clinical data from its partner iECURE's Phase 1/2 OTC-HOPE study. The first infant treated with ECUR-506, which uses Precision's ARCUS® nuclease technology, achieved a complete clinical response that was maintained from three to six months post-treatment.

The treatment was generally well-tolerated, with only asymptomatic transaminitis occurring at four weeks, which resolved within four weeks using immunosuppressive therapy. After twelve weeks, the patient no longer required ammonia scavenger medication and could increase protein intake to age-appropriate levels, with ammonia levels remaining normal.

iECURE expects to complete enrollment in 2025 and provide complete data in first half of 2026. The study is being conducted across the UK, US, Australia, and Spain. Separately, Precision's ELIMINATE-B trial for chronic hepatitis B is ongoing with Phase 1 data expected in 2025.

iECURE reported positive preliminary results from its Phase 1/2 OTC-HOPE clinical trial for ECUR-506, a gene editing therapy for ornithine transcarbamylase (OTC) deficiency. The first infant treated showed a complete clinical response at the lowest dose level, demonstrating successful removal of ammonia scavenging medicines and normalization of protein intake after three months, with results maintained through the six-month study period.

The treatment was generally well-tolerated, with only asymptomatic transaminitis occurring at four weeks, which resolved within four weeks through immunosuppressive therapy. The OTC-HOPE trial is currently enrolling patients across four regions (US, UK, Spain, and Australia), with complete data readout expected in 1H 2026. The therapy targets neonatal onset OTC deficiency, affecting over 1,000 births globally per year.

ECUR-506 utilizes two AAV vectors: an ARCUS® nuclease vector licensed from Precision BioSciences (DTIL) and a donor vector for OTC gene insertion.

Precision BioSciences (DTIL) has received Clinical Trial Application approval in Hong Kong for PBGENE-HBV, marking the first in vivo gene editing approach for chronic hepatitis B in Hong Kong and their second CTA approval in 2024. The ELIMINATE-B Phase 1 trial is actively recruiting patients in Moldova and expanding to Hong Kong.

PBGENE-HBV, utilizing the ARCUS® platform, is designed to cure chronic hepatitis B by eliminating cccDNA and inactivating integrated HBV DNA in hepatocytes. The therapy targets a viral site prevalent across all HBV genotypes, particularly relevant for Asia where over 400,000 individuals in Hong Kong live with chronic hepatitis B.

The company expects to report clinical data throughout 2025 and anticipates a U.S. investigational new drug (IND) application in 2025.

Precision BioSciences (Nasdaq: DTIL), a clinical stage gene editing company, has announced its participation in two upcoming investor conferences in November 2024. The company will take part in the JonesTrading Virtual Genetic Medicine Day on November 25 at 11:00 AM ET, participating in a panel discussion titled 'Next Generation of Gene Editing; Going Beyond "Cas"'. Additionally, Precision will present at AussieMit on November 29 at 2:50 PM AEDT, discussing 'Emerging Therapies'. The JonesTrading event webcast will be available on Precision's investor website, with replay access for approximately 30 days.

Precision BioSciences (DTIL) has presented preclinical data for PBGENE-HBV, its gene editing therapy for chronic hepatitis B, and outlined its Phase 1 ELIMINATE-B trial design. The preclinical data showed 99% viral DNA editing in non-human primates, effective hepatocyte distribution, and sustained declines in viral biomarkers. The ELIMINATE-B trial will evaluate up to 45 HBeAg-negative patients across five countries, with data expected throughout 2025. The company has received clearance to begin trials in Moldova, with additional regulatory applications pending. The therapy targets approximately 300 million people globally affected by chronic hepatitis B, addressing an unmet need in a multi-billion-dollar market where current treatments achieve functional cure in less than 3% of patients.

Precision BioSciences (Nasdaq: DTIL), a clinical stage gene editing company, announced it will release its financial results for the third quarter of 2024 on November 4, 2024. The company utilizes its proprietary ARCUS® platform to develop in vivo gene editing therapies aimed at sophisticated gene edits. Along with the financial results, Precision BioSciences will also provide a business update.

Precision BioSciences (Nasdaq: DTIL) announced a virtual Hepatitis B Investor Event scheduled for November 15, 2024. The event will feature Key Opinion Leaders and members of Precision's Hepatitis Scientific Advisory Board discussing the current hepatitis B treatment landscape, which only achieves 1-3% functional cure rates. The presentation will focus on PBGENE-HBV, their gene editing approach targeting cccDNA elimination and HBV DNA inactivation. The company will present final preclinical safety data and phase 1 clinical trial plans following Clinical Trial Application approval in Moldova.