[12/27/2024] Gene and Cell Therapy- weekly digest from PackGene

Featured

• Dec 23 | GEMMABio Pioneers Affordable Gene Therapies with $34M Seed Funding read more ?
• Dec 19 | A Phase 3 Ready DMD Gene Therapy, a Clinical Trial to Offset AAV Neutralizing Antibodies, and an AI Inspired New Generation of Capsids Highlight Genethon's 2024 Fall Milestones read more ?
• Dec 19 | TRYNGOLZA? (olezarsen) approved in U.S. as first-ever treatment for adults living with familial chylomicronemia syndrome as an adjunct to diet read more ?

News

• Dec 23 | Sumitomo Pharma America Announces U.S. FDA Approval of GEMTESA? (vibegron) for Men with Overactive Bladder Symptoms Receiving Pharmacological Therapy for Benign Prostatic Hyperplasia read more ?
• Dec 21 | YolTech Therapeutics Announces Successful Completion of Dose Escalation Phase in Phase I Trial of YOLT-201 for ATTR read more ?
• Dec 20 | ISCT Hails Landmark US FDA Approval of RYONCIL as Major Milestone for MSC Field read more ?
• Dec 20 | FDA Approves First Medication for Obstructive Sleep Apnea read more ?
• Dec 18 | Akamis Bio Raises $60 Million for Cancer Gene Therapy, Signs Licensing Deal with Xuanzhu Biopharma read more ?
• Dec 18 | DNA-Based Vaccine Production Provides Best Shot at Profitability read more ?

Articles

• Jan 28, 2025 | MIRO2 promotes cancer invasion and metastasis via MYO9B suppression of RhoA activity read more ?
• Jan 28, 2025 | Physiological shedding and C-terminal proteolytic processing of TMEM106B read more ?
• Jan 28, 2025 | Dual-nuclease single-cell lineage tracing by Cas9 and Cas12a read more ?
• Jan 28, 2025 | Dissociation-related behaviors in mice emerge from the inhibition of retrosplenial cortex parvalbumin interneurons read more ?
• Jan 28, 2025 | Causal contributions of cell-type-specific circuits in the posterior dorsal striatum to auditory decision-making read more ?
• Dec 24 | AAV-RNAi constructs promote DRG toxicity read more ?
• Dec 24 | Cutting through the stones: Unlocking therapeutic potential with gene editing tools for primary hyperoxaluria type 1 read more ?
• Dec 23 | Current and Emerging Issues in Adeno-Associated Virus Vector-Mediated Liver-Directed Gene Therapy read more ?
• Dec 20 | Choosing the right double-barreled gun: ARI0003 takes aim at lymphoma by targeting both CD19 and BCMA read more ?
• Dec 13 | Monitoring in vivo transcription with synthetic serum markers read more ?
• Dec 04 | From promise to practice: CAR T and Treg cell therapies in autoimmunity and other immune-mediated diseases read more ?