CRISPR Therapeutics, Inc.

Autolus Therapeutics announces FDA approval of AUCATZYL® (obe-cel) for adult B-cell Acute Lymphoblastic Leukemia, triggering $30m milestone payment from Blackstone. Obe-cel marketing authorizations are under review with MHRA and EMA. The company also appointed Matthias Will M.D. as Chief Development Officer and reported financial results for Q3 2024, with a net loss of $82.1m.

Capsida Biotherapeutics announced preclinical data for CAP-004, a gene therapy targeting CNS, cardiac, and sensory symptoms of Friedreich's ataxia. CAP-004 showed high transduction in key brain areas, significant cardiac tissue coverage, and potential for treating sensory vision loss, with a favorable safety profile.

Beam Therapeutics acknowledges a patient death in a Phase 1/2 trial for sickle cell disease, attributing it to pre-conditioning, not the base-editing treatment. AstraZeneca, UT, and LIVESTRONG report successful therapeutic genome editing in mouse liver via lipid nanoparticles. Korean researchers find base and prime editors produce fewer large DNA deletions than Cas9 nucleases. YolTech demonstrates effective mRNA delivery to bone marrow cells for blood disorder treatment. Japanese scientists develop a curative strategy for congenital purpura fulminans using engineered APC. US-France team identifies key host factors enhancing rotavirus vaccine production. Allogene Therapeutics shares positive Phase 1 data for renal cell carcinoma treatment. Cellectis presents strategies to enhance CAR T-cell efficacy in solid tumors. Caszyme and Integra Therapeutics enter a licensing agreement for safer gene therapies. Industry updates include financial results and clinical trial progress from various companies. CRISPR genome engineering symposium to feature latest advancements. CRISPR gene editing can integrate mitochondrial DNA into the nuclear genome, but exonucleases can mitigate this. HuidaGene Therapeutics' CRISPR-Cas13 RNA-editing therapy for macular degeneration is accepted by the FDA.

Two new gene editing medications for sickle cell disease, including the first FDA-approved CRISPR-based drug, face barriers like high costs and complex delivery processes. Pfizer withdrew its sickle cell drug Oxbryta due to safety concerns, and patient uptake for new gene therapies has been slow. Despite these challenges, the sickle cell treatment market is expected to grow, with over 40 companies developing more than 50 treatments. Cellarity is developing an oral drug to induce fetal hemoglobin, aiming to provide a more accessible treatment option.

Biotech and AI advances drive medicine's revolution with biologics surge, market growth, and precision medicine. Curative therapies, AI in drug discovery, and multiomics for early disease detection transform healthcare. CRISPR-based cures and AI-driven drug discovery reduce costs and time. Multiomics enhances cancer detection and treatment, improving survival rates and market expansion. ARK Genomic Revolution UCITS ETF offers exposure to these advancements.

Investor with 25+ years of experience emphasizes compounding, dividend reinvesting, and patient investing. Believes in slow accumulation of high-quality assets mixed with high-risk opportunities. Invests with integrity, focusing on companies improving the world. Self-taught, reads widely, and teaches at the university level. Holds beneficial long positions in CRSP, VRTX shares.

Vertex Pharmaceuticals developed a therapy for cystic fibrosis, challenging low drug development odds. Their R&D strategy focuses on causal human biology, using human genomics and modality agnosticism to improve success rates, exemplified by FDA approvals for CF, sickle cell disease, and beta thalassemia treatments.

Pfizer's withdrawal of Oxbryta from global markets due to increased risk of deaths and complications has left the sickle cell disease community reeling. Despite recent setbacks, hope for SCD treatment lies in next-gen transplantation and gene therapy, with St. Jude developing its own gene therapy. Pfizer's inclacumab and osivelotor, Agios Pharmaceuticals' mitapivat, Novo Nordisk's etavopivat, and Fulcrum Therapeutics' pociredir are among investigational therapies in the SCD pipeline.

Breakthrough Properties has launched Torrey View, a 520,000-square-foot life science campus in San Diego, fully preleased and LEED Gold certified. Pfizer and Becton, Dickinson and Co. are major tenants, with Charles River Laboratories and others also occupying space. The campus features various amenities and is part of Breakthrough's portfolio of over 5 million square feet of life science space in operation or development.

CRISPR Therapeutics and Moderna, despite share declines, are strong long-term investments due to CRISPR's Casgevy approval and Moderna's diverse mRNA pipeline.