August 2026– date –
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BMS’s First CELMoD Therapy ZENBEXUS™ Receives Accelerated FDA Approval in Combination with Daratumumab for Multiple Myeloma, Including Early Relapse Patients
Bristol Myers Squibb USA Overview The U.S. FDA granted accelerated approval to Bristol Myers Squibb's first Cereblon E3 ligase modulator (CELMoD) therapy, ZENBEXUS™ (iberdomide), in combination with daratumumab, hyaluronidase-fihj, and d... -
FDA Grants Accelerated Approval to Replimune’s Onco-Viral Therapy Tudriqev in Combination with Nivolumab for Advanced Melanoma, Offering New Treatment Option
U.S. Food and Drug Administration USA Overview The U.S. FDA granted accelerated approval to Replimune, Inc.'s oncolytic viral therapy Tudriqev (vusolimogene oderparepvec-wtpg) in combination with nivolumab for adult patients with advance... -
Ractigen Therapeutics Completes Phase II Enrollment for SOD1-ALS siRNA RAG-17; LiCO Therapy RAG-18 for DMD Receives Rare Pediatric Disease Designation
Facebook China Overview Ractigen Therapeutics announced the completion of patient enrollment and first dosing in its Phase II clinical trial for RAG-17, an investigational siRNA therapy for SOD1-mutated amyotrophic lateral sclerosis (ALS... -
Gilead Report Highlights RNA Therapeutic Progress: GSK/Ionis HBV ASO Shows Positive Phase 3, Novartis FSHD siRNA Promising Early Data
The Bio Report USA Overview A Gilead report underscores significant advancements in RNA therapeutic clinical trials, with GSK/Ionis's beoprovirsen, an ASO targeting HBV, demonstrating positive Phase 3 results. Novartis's siRNA therapy, D... -
Denali Therapeutics Advances DNL628 (MAPT-Targeting ASO) for Neurodegenerative Diseases, DNL593 for GRN FTD Granted Orphan Drug Designation
Seeking Alpha USA Overview Denali Therapeutics is progressing DNL628, an intravenously administered ASO designed to reduce tau by targeting MAPT, with initial Phase 1b biomarker data expected in H1 2027 from its TransportVehicle platform... -
SynaptixBio Accelerates Regulatory Pathway for Ultra-Rare ASO Drugs, Expanding Therapeutic Potential of TUBB4A Mutant Silencers
Advancing RNA USA Overview SynaptixBio is effectively navigating the regulatory landscape for ultra-rare antisense oligonucleotide (ASO) drugs, securing Orphan Drug and Rare Pediatric Disease designations in collaboration with the FDA, E... -
Comprehensive Guide Published: RNA Therapeutics, from ASO to LNP/GalNAc Platforms, Shaping the Future of Precision Medicine
MDPI International Overview A comprehensive guide published by MDPI overviews the engineering history of RNA therapeutics, spanning from ASOs to modern siRNA and mRNA-LNP-based therapies. It highlights a growing regulatory trend favoring... -
Japan Accelerates Next-Gen mRNA Vaccine and Therapeutic Development: saRNA and circRNA Boost Immunity and Efficiency
MJA InSight Australia Overview Research into next-generation mRNA vaccines, particularly self-amplifying RNA (saRNA) and circular RNA (circRNA), is rapidly advancing, focusing on enhancing immune durability and efficiency. Beyond infecti... -
Wave Life Sciences’ Obesity Drug WVE-007 Achieves 15% Visceral Fat Reduction in Phase 1, Advancing to Phase 2; AATD RNA Editing Therapy WVE-006 Anticipates Accelerated Approval
BigGo Finance USA Overview Wave Life Sciences' experimental siRNA therapy, WVE-007, successfully reduced visceral fat by 15% after a single injection in Phase 1 trials and has initiated Phase 2a. This drug targets the INHBE gene in the l... -
FDA Approves Dawnzera: RNA-Targeting ASO Marks Paradigm Shift for Hereditary Angioedema
Liv Hospital Turkey Overview In August 2025, the FDA granted approval to Dawnzera (donidalorsen-azyh), a groundbreaking RNA-targeting antisense oligonucleotide (ASO) for hereditary angioedema (HAE). By specifically silencing prekallikrei...
