Allogene Therapeutics

Allogene Therapeutics' Q3 2024 earnings call highlighted the ALPHA3 trial for cema-cel, ALLO-316's 50% best overall response rate in renal cell carcinoma, and ALLO-329's IND filing in Q1 2025. Despite a $66.3 million net loss, Allogene maintains a $403.4 million cash balance with a runway into 2026, focusing on CAR T therapies for cancer and autoimmune diseases.

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.

ALLO-316, an allogeneic CAR T-cell therapy, received FDA's RMAT designation for treating CD70-positive advanced or metastatic RCC. Data from the TRAVERSE trial showed an ORR of 17% and DCR of 89% in all evaluable patients, with higher rates in CD70-positive patients. Safety data indicated AE profiles consistent with autologous CAR T-cell therapies, with one DLT of grade 3 autoimmune hepatitis. The ongoing TRAVERSE trial continues to enroll RCC patients who have received prior immune checkpoint inhibitor and VEGF inhibitor treatments.

Legend Biotech to open R&D center in Philadelphia, employing 55 staff, highlighting the city's growing biotech hub status.

FDA grants RMAT designation to ALLO-316 for treating advanced or metastatic CD70 positive RCC, based on TRAVERSE trial data. ALLO-316 aims to advance Dagger® technology as a next-gen allogeneic platform for 'off-the-shelf' CAR T therapy in solid tumors.

The FDA granted a Regenerative Medicine Advanced Therapy (RMAT) designation to ALLO-316, an AlloCAR T product for advanced RCC patients who have received prior immune checkpoint inhibitor and VEGF-targeting therapy. The RMAT designation is supported by interim data from the phase 1 TRAVERSE trial, showing a disease control rate of 89% and an overall response rate of 17% among 18 RCC patients. The safety profile of ALLO-316 is consistent with other CAR T therapies, with neurotoxicity and cytokine release syndrome as common adverse events.

Artiva Biotherapeutics appoints Alison Moore, Ph.D., as independent Board member, leveraging her 25+ years in biotech and pharma, focusing on cell therapy manufacturing.

The gene therapy market is rapidly expanding, with over 180 companies developing more than 200 gene therapies. Key players include Ultragenyx Pharmaceutical Inc, Rocket Pharmaceuticals, and Adverum Biotechnologies. Prominent therapies in the pipeline include DTX401, RP-L102, and ADVM-022. Gene therapy aims to treat diseases by modifying genes, offering potential long-lasting effects with fewer side effects. Challenges include immune reactions, long-term effects, and high costs.