Beam Therapeutics

CRISPR Therapeutics' stock is down 47% since March due to slow Casgevy launch, despite FDA and E.U. approvals for treating SCD and TDT. The company has five other clinical-stage therapy candidates, with CTX112 showing promising results in blood cancer treatment. Despite current losses, CRISPR's $1.9 billion cash reserves and partnership with Vertex provide a long runway for potential growth, though the stock remains a high-risk investment.

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.

A patient in Beam Therapeutics’ BEACON trial for sickle cell disease died due to respiratory failure after busulfan conditioning, not BEAM-101 therapy. Beam will present updated BEACON data and preclinical ESCAPE approach at ASH 2024, which avoids busulfan conditioning using a CD117 monoclonal antibody, showing promise in nonhuman primates.

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.

Beam Therapeutics reported a patient death in a Phase I/II trial for BEAM-101, attributing it to the preconditioning regimen, not the treatment. The FDA allowed the trial to continue without changes. Researchers seek less toxic alternatives to busulfan, crucial in cell and gene therapy protocols.

Beam Therapeutics reports a patient death in BEAM-101 trial for sickle cell disease, unrelated to the therapy. Sana Biotechnology refocuses on autoimmune assets, reducing workforce. Ring Therapeutics expands in Singapore with new deals. Drug regulators criticized for ignoring forced labor in supply chains. OSE Therapeutics' IL-7 therapy shows positive results in ulcerative colitis trial. Japan's PMDA opens second overseas office in Washington.

Beam Therapeutics' CRISPR-based gene editing therapy for sickle cell disease showed success in early trials, but a patient's death linked to busulfan, a chemotherapy drug used in the treatment, raises safety concerns. Beam is developing an alternative using an antibody to avoid busulfan's toxicity, with promising preclinical results in monkeys. Early data from BEAM-101 shows increased fetal hemoglobin levels and reduced sickled hemoglobin, potentially alleviating sickle cell symptoms.

Apellis Pharmaceuticals reports $196.8 million Q3 2024 revenue, driven by SYFOVRE and EMPAVELI sales. The company faces market, operational, and regulatory risks, including a CHMP negative opinion on SYFOVRE in Europe. Apellis plans to submit FDA and EMA applications for systemic pegcetacoplan in 2025, and is developing APL-3007 and an oral complement inhibitor.

The red biotechnology market is projected to grow from $509.71 billion in 2023 to $867.20 billion by 2028, driven by advancements in personalized medicine, biosimilars, and genetic engineering. Major players include Pfizer, Roche, Bayer, and Amgen.