Precision BioSciences

BMO Capital upgraded Precision BioSciences to Outperform, citing promising clinical data for its gene editor, Arcus, and a favorable risk/reward at current share levels, with a $34 price target.

ECUR-506, an in vivo gene editing therapy for OTC deficiency, showed complete clinical response in a Phase 1/2 trial, with no significant safety issues. The treatment allowed discontinuation of ammonia scavenger medication and increased protein intake, maintaining normal ammonia levels. The OTC-HOPE study continues, with full data expected by 2026.

Precision Biosciences gains CTA approval in Hong Kong for PBGENE-HBV, a gene editing programme targeting hepatitis B viral genome in ELIMINATE-B Phase I trial. The company is recruiting subjects in Moldova and activating a site in Hong Kong, utilizing the ARCUS platform for precise gene editing. Precision plans to file a new US IND next year and continue updates on regulatory approvals and treatment commencement.

Precision BioSciences received approval in Hong Kong for PBGENE-HBV Phase 1 ELIMINATE-B trial, targeting chronic hepatitis B. The trial aims to eliminate cccDNA and inactivate integrated HBV DNA, potentially leading to functional cures. The study is global and multi-site, with recruitment ongoing, and expected to report data throughout 2025. A U.S. IND is anticipated in 2025.

Precision BioSciences receives Hong Kong CTA approval for PBGENE-HBV, enabling the ELIMINATE-B Phase 1 trial expansion to treat chronic hepatitis B. The trial, using the ARCUS® platform, aims to eliminate cccDNA and inactivate HBV DNA, with clinical data expected throughout 2025 and a U.S. IND anticipated in 2025.

The CRISPR and Cas gene market was valued at $3.3 billion in 2023, with a CAGR of 20.38% since 2018. It's projected to reach $8.8 billion by 2028 and $24.6 billion by 2033. Growth drivers include personalized medicine, genetic disorders, and synthetic biology. Challenges include regulatory complexities and healthcare access in developing countries. The market is segmented by type, application, product type, end use, and service type, with various segments showing significant growth potential. North America leads the market, but Asia-Pacific and Africa are expected to grow fastest. The market is dominated by large players like Danaher Corporation and Agilent Technologies Inc.

The gene editing market is projected to grow from USD 4.66 billion in 2024 to USD 7.59 billion by 2029, driven by advancements in technologies and expanding applications. The products segment, including kits, reagents, libraries, and software, dominates the market. CRISPR technology leads, while Asia Pacific shows the highest growth rate due to increased R&D and government funding. Challenges include off-target effects and scalability issues.

AffyImmune Therapeutics appoints Deyaa Adib, M.D., as Chief Medical Officer, bringing 28 years of oncology experience. Adib will oversee clinical activities, including strategic direction and execution of development programs. He has led successful registrations in solid tumor and hematologic malignancy indications and has held CMO roles at FibroGen, Triumvira Immunologics, Rain Therapeutics, and Blueprint Medicines.

Precision BioSciences received approval from Moldova's health authorities to conduct a clinical trial for PBGENE-HBV, an in vivo gene editing therapy targeting chronic hepatitis B. The therapy aims to eliminate the viral reservoir, potentially offering a functional cure. Precision BioSciences plans to expand the trial globally, with a focus on regions with lower vaccination coverage. Political instability in Moldova could impact the trial's regulatory framework and collaboration with international bodies.

Merck licenses a new cancer drug from LaNova Medicines for $588 million upfront and up to $2.7 billion in milestone payments. GOP-controlled Congress gives Trump broad power over health care policies. Vertex Pharmaceuticals plans to announce results from a mid-stage trial for sciatica treatment by year-end. CDC and FDA officials warn of anti-vaccine consequences. Tune Therapeutics to begin human testing of a gene-editing therapy for hepatitis B. Amgen defends its obesity drug amid bone concerns. Neurogene criticized for not conducting randomized, placebo-controlled trials for its Rett syndrome gene therapy.