Intellia Therapeutics

Intellia Therapeutics announces positive Phase 1 data for nex-z, a CRISPR-based gene editing therapy for ATTR amyloidosis, showing rapid, deep, and durable serum TTR reduction and favorable safety profile. Data presented at the 2024 AHA Scientific Sessions and published in the New England Journal of Medicine.

Intellia Therapeutics announced positive Phase 1 data for nexiguran ziclumeran (nex-z), a CRISPR-based gene editing therapy for ATTR amyloidosis, showing rapid, deep, and durable serum TTR reduction with disease stabilization or improvement. ATTR-CM patients had a 90% mean serum TTR reduction at 12 months, and ATTRv-PN patients had a 91% reduction, both with favorable safety profiles.

Intellia Therapeutics announces positive Phase 1 data for nex-z in ATTR amyloidosis, showing rapid, deep, and durable serum TTR reduction, disease stabilization or improvement, and favorable safety profile. Data presented at 2024 AHA Scientific Sessions and published in NEJM.

CRISPR technology market to grow at 19.2% CAGR (2024-2031), driven by biomedical applications and increased R&D funding. Key players include Merck KGaA, Thermo Fisher Scientific, and Beam Therapeutics, focusing on genetic disorder treatments like sickle cell disease and Alpha-1 Antitrypsin Deficiency.

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.

Brendan, a Ph.D. in organic synthesis from Stanford, worked at Merck and co-founded 1200 Pharma at Caltech. He remains an investor, focusing on biotech stocks. NTLA is a high-risk investment, akin to a long-dated call option, with potential for substantial gains or losses based on the success of NTLA-2001 and 2002.

Acoramidis (Attruby; BridgeBio) approved by FDA for transthyretin amyloid cardiomyopathy (ATTR-CM), based on ATTRibute-CM trial results. BridgeBio committed to providing the drug free for life to trial participants, while the drug's $244,000/year price draws scrutiny. Acoramidis mimics a natural TTR gene mutation, targeting ATTR-CM's root cause, and BridgeBio plans to seek approval in Europe, Japan, and Brazil. Additionally, gene-editing therapy nexiguran ziclumeran (nex-z; Intellia Therapeutics) shows promise in early trials for ATTR-CM.

NTLA-2002, an in vivo CRISPR-Cas9 gene-editing therapy targeting the KLKB1 gene in HAE patients, showed a single dose reduced angioedema attacks and kallikrein levels, supporting its potential as a functional cure. The therapy was well-tolerated with mild-to-moderate AEs, and Intellia plans to use the 50 mg dose in its phase 3 HAELO trial.

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.

HuidaGene Therapeutics' HG202, a Cas13-based RNA-editing therapy for neovascular age-related macular degeneration (nAMD), received FDA approval for clinical trials. HG202 aims to treat nAMD patients resistant to anti-VEGF treatment, with pre-clinical studies showing significant reduction in CNV area. The BRIGHT trial will assess HG202's safety and tolerability in nAMD patients.