Oligonucleotide Therapeutics and Delivery Conference

U.S. FDA Approves ZEVASKYN™ (prademagene zamikeracel), the First-Ever Cell-Based Gene Therapy for Recessive Dystrophic Epidermolysis Bullosa (RDEB) Patients

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Lexeo Therapeutics Reports Positive Interim Phase 1/2 Results for LX2006 in Friedreich’s Ataxia Cardiomyopathy, Paving the Way for Registrational Trial

Lexeo Therapeutics reported positive interim Phase 1/2 data for LX2006 in Friedreich’s ataxia cardiomyopathy, showing improved cardiac function and increased frataxin levels, supporting advancement to a registrational studyReadmore

Rznomics Unveils World’s First RNA Editing Therapy, Aiming to Make TSR the Standard of Care

Rznomics is advancing anticancer and rare disease therapies using its RNA editing platform, TSR, and aims for KOSDAQ listing by next year. Readmore

Gene therapy shows promise for treating rare muscle disease

U of A researchers develop a molecule that bypasses genetic mutation to restore a crucial muscle-repairing protein. Readmore

NeuroSense Therapeutics Reports Promising Phase 2b microRNA Results, Showcasing PrimeC’s Potential as a Disease-Modifying ALS Therapy

NeuroSense Therapeutics (NRSN) reported encouraging results from its Phase 2b PARADIGM clinical trial, emphasizing the notable effects of its investigational ALS therapy, PrimeC, on microRNA (miRNA) modulation. Readmore

Scientists Deploy Nanoparticles to Combat Glioblastoma in Mice

In a recent study, researchers from the University of Michigan developed nanodiscs that target cholesterol levels in GBM cells, effectively starving the cancer and improving survival rates in treated mice.Readmore

Atelerix has entered into an exclusive agreement with MineBio to create a distribution channel for its non-cryogenic cell preservation solutions in China.

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Avidity Biosciences Announces Inducement Grants in Accordance with Nasdaq Listing Rule 5635(c)(4)

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Creyon Bio names seasoned industry executive Serge Messerlian as CEO, drawing on his decades of experience in company building and strategic partnerships.

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PepGen Shares Latest Developments in CONNECT Program

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Best Technologies Innovation Award Presented to Asahi Kasei Bioprocess America at INTERPHEX 2025

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Biparatopic Bispecific Antibody-Drug Conjugate (ADC) Platform Targeting c-MET

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Creyon Bio and Lilly Announce Collaboration to Develop RNA-Targeted Oligonucleotide Therapies

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Atalanta Therapeutics Releases Preclinical Data Backing RNAi Therapy for KCNT1-Related Epilepsy

Atalanta Therapeutics announced preclinical results in Epilepsia showing long-lasting seizure reduction and behavioral improvements with its RNAi therapy, ATL-201, in a KCNT1-related epilepsy model. An IND filing is planned for 2025Readmore

AlphaRose Therapeutics Announces Acquisition of Alpha Anomeric SA, a French Leader in Oligonucleotide Technology

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Regulus Therapeutics Announces Agreement for Acquisition by Novartis AG

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BioMarin Announces First Quarter 2025 Financial Results and Confirms Full-Year Outlook

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Praxis Precision Medicines, Inc. Announces Inducement Grants in Accordance with Nasdaq Listing Rule 5635(c)(4)

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HAYA Therapeutics to Showcase RNA-Guided Regulatory Genome Platform at the American Society of Gene & Cell Therapy (ASGCT) Annual Meeting

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Isarna Therapeutics Unveils Positive Final Phase 2 BETTER Trial Results at ARVO 2025

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CRISPR Therapeutics and Sirius Therapeutics Announce Strategic Collaboration to Develop Innovative Multi-Target siRNA Therapies

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Rznomics Signs Global Licensing Deal with Eli Lilly for RNA-Editing Therapies

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In-Utero Injection into Amniotic Fluid Halts Spinal Muscular Atrophy Progression

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TriLink BioTechnologies® Launches First mRNA Synthesis Kit Featuring CleanCap Technology, Donates Kits to Leading Academic Institutions

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Ionis Reports Positive Topline Results from ESSENCE Study Evaluating Olezarsen in Individuals with Moderately Elevated Triglycerides

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Shift Bioscience Announces Appointment of Lord David Prior and Sir Tony Kouzarides to Advance Long-Term Strategy in Cell Rejuvenation Therapeutics

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An ancient RNA-guided system could simplify delivery of gene editing therapies

A vast search of natural diversity has led scientists at MIT’s McGovern Institute for Brain Research and the Broad Institute of MIT and Harvard to uncover ancient systems with potential to expand the genome editing toolbox. Readmore

Ionis and Ono announce global license agreement for sapablursen in polycythemia vera

Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) and Ono Pharmaceutical Co., Ltd. (Ono) have entered into a new partnershipReadmore

Canada’s first RNA trial for rare ataxia begins at The Neuro.

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Uncovering New Therapeutic Targets for Pediatric Epilepsy

Researchers in the laboratory of Dr. Gemma Carvill at Northwestern University have uncovered new insights into Dravet syndrome, a rare and severe form of childhood epilepsy. Readmore

Regulus Therapeutics Announces Fourth Quarter and Year-End 2024 Financial Results and Business Updates

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ProQR Therapeutics Moves Forward with Axiomer RNA Editing Pipeline, Setting Key Milestones for 2025

ProQR Therapeutics, a company committed to transforming lives through innovative RNA therapies, has shared its financial results for 2024 along with key business updates.Readmore

Australian-first partnership to develop mRNA medicines for autoimmune diseases

In an Australian-first, WEHI has joined forces with biotech company Moderna to advance the development of mRNA medicines for autoimmune diseases.Readmore

NIH cuts research grants on vaccine hesitancy—with mRNA vaccine studies the next target

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RPI Researchers Aim to Manufacture mRNA More Affordably

A sponsored research agreement with BioNTech will fund a project that aims to improve the mRNA manufacturing processReadmore

Groundbreaking study shows potential of new mRNA vaccine to help fight tuberculosis

A new vaccine that boosts immunity against tuberculosis (TB) has been shown to be effective in pioneering pre-clinical trials, as part of a successful collaboration between three leading Australian research institutions. Readmore

Amylyx Pharmaceuticals has announced the dosing of the first participant in the Phase 1, Multiple Ascending Dose LUMINA trial of AMX0114 in individuals with Amyotrophic Lateral Sclerosis (ALS)

Amylyx Pharmaceuticals announced the dosing of the first participant in LUMINA, a Phase 1, multinational trial of AMX0114, an investigational antisense oligonucleotide targeting calpain-2 in ALS patients. Early cohort data is expected in 2025Readmore

Nosis Biosciences Announces Research Collaboration and Option Agreement with Daiichi Sankyo to Develop Cell-Targeted RNA Medicines

Nosis Biosciences, an AI-driven biotechnology company specializing in RNA therapeutics for challenging cell types, today announced it entered into a Research Collaboration and Option Agreement with Daiichi SankyoReadmore

Rona Therapeutics Receives IND Clearance from FDA to Initiate Phase 2 Study of RN0361: an APOC3 Targeted siRNA for Management of Hypertriglyceridemia

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Biogen’s Investigational Tau-Targeting Therapy BIIB080 Earns FDA Fast Track Designation for Alzheimer’s Disease Treatment

Biogen Inc. announced that the U.S. Food and Drug Administration has granted Fast Track designation to BIIB080, its investigational antisense oligonucleotide therapy targeting tau for Alzheimer’s disease. Readmore

Avidity Biosciences has been granted Orphan Drug Designation in Japan for Delpacibart Etdesiran (del-desiran) for the treatment of Myotonic Dystrophy Type 1

Avidity Biosciences' delpacibart etedesiran (del-desiran) has received Orphan Drug Designation in Japan for treating myotonic dystrophy type 1 (DM1), the first DM1 treatment to do so. It also holds similar designations from the FDA and EMA. Readmore

A cellular regulator of mRNA vaccines has been discovered, opening up new therapeutic possibilities

Dr. Kim V. Narry's team at the Institute for Basic Science has revealed a key cellular mechanism affecting mRNA vaccine function, offering insights to improve RNA-based treatments.Readmore

Ractigen's RAG-17 ALS Data Impresses at AAN 2025: Wins Top Award, Phase I Progresses

Ractigen Therapeutics announced that the final positive results from the Investigator-Initiated Trial (IIT) of RAG-17, a siRNA therapy for ALS targeting SOD1, were presented for the first time at the AAN 2025 Annual Meeting in San Diego. Readmore

Engineering Efficient Delivery Methods for Gene Editing

A team from Helmholtz Munich and the Technical University of Munich has developed ENVLPE, an advanced delivery system that uses engineered virus-like particles to efficiently transport CRISPR/Cas9 gene-editing tools into cells.Readmore

New Gene Editing Tool Shows Promise for Treating Diseases with Multiple Mutations

Scientists at Mass General Brigham and Beth Israel have developed STITCHR, an RNA-only gene editing tool that inserts full genes precisely without unwanted mutations. Readmore

Entos collaborates with Circio for in vivo PLV-circVec delivery

Circio and Entos have partnered to develop and test in vivo delivery of circular RNA using Entos’ Fusogenix™ PLV™ technologyReadmore

Solu Therapeutics Secures $41M in Series A Funding and Begins Dosing First Patient in Phase 1 Trial of STX-0712 for CMML and Advanced Blood Cancers

Solu Therapeutics announced the close of a $41M Series A round, with new backing from investors including Eli Lilly, Biovision Ventures, and The Leukemia & Lymphoma Society. Readmore

Serina Therapeutics Raises $5 Million to Advance SER-252 into Clinical Trials for Advanced Parkinson's Disease

Serina Therapeutics, Inc., a clinical-stage biotechnology company specializing in its proprietary POZ Platform™ drug optimization technology, has closed a $5 million financing round with support from strategic shareholders. Readmore

The winner of the 2025 research prize has been announced: The 2025 Else Kröner Fresenius Prize for Medical Research is awarded to RNA therapeutics for the treatment of Huntington's disease.

Anastasia Khvorova, PhD, RNA therapeutics professor at UMass Chan Medical School, will receive the 2025 Else Kröner Fresenius Prize for Medical Research on May 15, 2025. Readmore

CureVac has received U.S. FDA IND clearance to begin a Phase 1 clinical trial for its novel mRNA-based precision immunotherapy targeting squamous non-small cell lung cancer

CureVac announced that the U.S. FDA has cleared its IND application for a Phase 1 clinical study of CVHNLC in squamous non-small cell lung cancer patients. The study will explore the potential of CureVac's mRNA-based therapy in this patient group. Readmore

ABL Bio has announced a license agreement with GSK for its Grabody-B brain delivery platform, aimed at developing novel medicines for neurodegenerative diseases.

ABL Bio and GSK have entered a multi-program agreement to develop medicines for neurodegenerative diseases using ABL Bio’s Grabody-B platform. ABL Bio will receive up to £77 million in upfront and near-term payments. Readmore

Silexion Therapeutics Reports Groundbreaking Positive Initial Data from Systemic Administration of SIL204 in Orthotopic Pancreatic Cancer Models

Silexion Therapeutics' Latest Data Demonstrates that Subcutaneously Administered SIL204 Reduces Both Primary Tumors and Metastases in Clinically Relevant Orthotopic Models Where Human Pancreatic Cancer Cells Grow in Their Native EnvironmentReadmore

Capricor Therapeutics Announces First Quarter 2025 Financial Results and Provides Corporate Update

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Hoth Therapeutics Announces Encouraging Preclinical Results for HT-KIT, a Precision Antisense Therapy for Rare and Aggressive KIT-Driven Cancers

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Editas Medicine Releases First Quarter 2025 Earnings and Operational Highlights

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Secarna Pharmaceuticals and Curie.Bio Portfolio Company Sign Research and Option Agreement

Secarna Pharmaceuticals GmbH & Co. KG, a leader in the discovery and development of next-generation oligonucleotide therapeutics, has announced the signing of a research and option agreement with a portfolio company of Curie.Bio. Readmore

Amylyx Pharmaceuticals Announces Financial Results for the First Quarter of 2025

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Sarepta Therapeutics Reports First Quarter 2025 Financial Results and Corporate Highlights

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Vertex Pharmaceuticals has announced its financial results for the first quarter of 2025.

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