Gene Editing Breakthrough: David Liu's Base and Prime Editing Technologies Transform Treatment of Genetic Diseases
• David Liu, Harvard professor and Broad Institute researcher, has been awarded the Breakthrough Prize for developing base and prime editing technologies that correct DNA mutations without cutting the double helix.
• Liu's gene editing technologies have been distributed to over 20,000 non-profit labs worldwide and are currently being tested in 15 clinical trials across five countries, showing promising results for rare genetic diseases.
• The technology has already demonstrated life-saving outcomes in patients with T-cell leukemia, sickle-cell disease, beta-thalassemia, and high cholesterol, making treatments for rare genetic diseases economically feasible.
Beam Therapeutics Achieves Breakthrough in Base Editing for Alpha-1 Antitrypsin Deficiency
• Beam Therapeutics' BEAM-302 demonstrated successful DNA correction in alpha-1 antitrypsin deficiency patients, marking the first clinical proof of concept for direct mutation correction using base editing technology.
• The treatment increased properly folded AAT protein levels up to 2.8 times baseline with a 78% reduction in misfolded protein in one high-dose patient, potentially addressing both liver and lung manifestations of the disease.
• Initial safety data from the nine-patient trial appears favorable, with the company now planning to test higher doses and expand enrollment to include patients with mild-to-moderate liver disease.
ACTG Launches ACACIA Trial in Africa to Evaluate HIV Cure Strategy
• The ACTG has initiated the ACACIA (A5417) study, a Phase 2 trial, to assess the safety and efficacy of broadly neutralizing antibodies (bNAbs) in HIV treatment.
• ACACIA will evaluate the combination of 3BNC117-LS and 10-1074-LS with antiretroviral therapy (ART) in adults living with HIV across four African countries.
• The trial includes an analytic treatment interruption (ATI) phase to determine if the intervention can control HIV in the absence of ART, a key step in HIV cure research.
• ACACIA aims to optimize the therapeutic benefit of bNAbs by administering them when viral load is high and assessing their long-lasting immunologic effects.
Leptin-GLP-1 Combination Shows Promise in Obesity Treatment
• A novel obesity drug combination using leptin, a hormone discovered 30 years ago, with GLP-1 drugs shows promising results in new data published in Science Translational Medicine.
• Initial hopes for leptin as an obesity treatment were unsuccessful, but new research casts the hormone in a new light when combined with GLP-1 drugs.
• The leptin and GLP-1 combination could offer a new approach to obesity treatment by targeting body fat regulation.
Novel Enzyme Shows Promise in Treating IgG-Mediated Autoimmune Diseases
• Researchers at Emory University have identified a novel enzyme, CU43, that effectively reduces IgG-mediated pathologies in mouse models of autoimmune diseases.
• The enzyme demonstrated remarkable potency, requiring 4,000 times less dosage than current treatments for myasthenia gravis to achieve similar symptom reduction.
• This new enzyme could potentially offer fewer side effects and alternative administration options for patients with a range of autoimmune disorders.
• The research team plans to expedite clinical trials in humans to explore the enzyme's therapeutic potential across various IgG-mediated pathologies.
Artax Biopharma's AX-158 Demonstrates Clinical Validation in Phase 2a Psoriasis Trial
• Artax Biopharma's AX-158, a first-in-class oral Nck modulator, has achieved clinical validation in a Phase 2a trial for psoriasis, showcasing a novel approach to autoimmune disease treatment.
• The Phase 2a study demonstrated statistically significant improvements in T cell and psoriasis-related biomarkers, aligning with observed PASI improvements in patients with mild to moderate plaque psoriasis.
• AX-158 exhibited a well-tolerated safety profile in the trial, with no reported infections or discontinuations, reinforcing its potential as a safe and effective treatment option.
• Further studies in other autoimmune diseases, such as atopic dermatitis, are planned, with clinical results anticipated by the end of 2026, expanding the potential applications of Nck modulation.
Why BET Inhibitors Fail in Estrogen-Driven Breast Cancer and How to Improve Them
• A new study explains why BET inhibitors, promising for other cancers, are ineffective against estrogen receptor-positive breast cancer, revealing they only partially block oncogene activation.
• Researchers found that cancer-related genes depend on the BET protein itself, not just its bromodomain, and BRD4 can activate MYC without relying on its bromodomain.
• The study identified that BRD4 interacts with the Mediator complex, crucial for gene transcription and estrogen receptor function, independent of the bromodomain.
• Scientists are now focusing on a more thorough understanding of the BRD4-Mediator complex structure and exploring combination therapies to target both the bromodomain and transcriptional backdoors.
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