Oligonucleotide Therapeutics and Delivery Conference

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

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

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

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

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

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

New RNA and protein tools may improve cell therapies

Two new papers demonstrate novel ways of fine-tuning cellular functions, with applications in cancer treatment, diagnostics, and more 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

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

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

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

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

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

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

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

Biogen's investigational tau-targeting therapy, BIIB080, has received FDA Fast Track designation for the treatment of Alzheimer's disease.

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

GreenLight Biosciences Announces Close of Additional Fundraising

Just Climate’s $25 Million Investment Supports Global Expansion and Commercialization of Breakthrough RNA Technology Readmore

Singapore launches S$130 million initiative in RNA research

A new national programme that aims to position Singapore at the forefront of advancements in RNA science and applications was officially launched by Singapore Deputy Prime Minister Mr Heng Swee Keat, who is also Chairman of National Research Foundation. Readmore

New S$130 million national research initiative to enhance Singapore’s strategic research capabilities in RNA biology and its applications

A new national programme that aims to position Singapore at the forefront of advancements in RNA science and applications was officially launched on 24 March 2025. Readmore

Scientists gain insight into RNA-editing protein that could lead to improved treatment for cancer, autoimmune diseases

A research team led by Rice University’s Yang Gao has uncovered new insights into the molecular mechanisms of ADAR1, a protein that regulates ribonucleic acid (RNA) induced immune responses. Readmore

Gene therapy shows promise for treating rare muscle disease

In newly published research findings, A University of Alberta research team reports it has developed a new antisense oligonucleotide to treat dysferlinopathy. Readmore

Secarna Pharmaceuticals will present new positive data on its antisense oligonucleotide SECN-15, targeting NRP1, both as a monotherapy and in combination with immune checkpoint inhibitors, at AACR.

The data will highlight SECN-15's potential to substantially enhance the efficacy of immune checkpoint inhibitors. Secarna is preparing to begin first-in-human trials in mid-2026 to further investigate this promising combination therapeutic approach. Readmore

Novotech has released the 2025 Global Clinical Trial Landscape Report on Hepatitis B.

Novotech, a global full-service clinical Contract Research Organization (CRO), has released its 2025 Hepatitis B Global Clinical Trial Landscape report. Readmore