Antisense oligonucleotides - Articles - Page 3

Neurology/Psychiatric

Vanda's ASO VCA-894A awarded US orphan drug designation for Charcot-Marie-Tooth disease type 2S

June 2, 2023

Vanda Pharmaceuticals Inc.'s antisense oligonucleotide (ASO) VCA-894A has been awarded orphan drug designation by the FDA for the treatment of Charcot-Marie-Tooth disease, axonal, type 2S (CMT2S), caused by cryptic splice site variants within IGHMBP2.

Ocular

Stoke Therapeutics’ ASO STK-002 cleared to enter clinic in U.K. for autosomal dominant optic atrophy

April 26, 2023

No Comments

Stoke Therapeutics Inc. has received clinical trial application (CTA) clearance from the U.K.’s Medicines and Healthcare products Regulatory Agency (MHRA) to initiate a phase I/II study (OSPREY) of STK-002 for the treatment of autosomal dominant optic atrophy (ADOA).

Neurology/Psychiatric

BMD-001, a nanoparticle-formulated ASO with efficacy in preclinical models of Parkinson’s disease

April 12, 2023

No Comments

Researchers from Biorchestra Ltd. presented preclinical data for BMD-001, a novel nanoparticle-formulated antisense oligonucleotide (ASO) being developed for the treatment of Parkinson’s disease (PD). miR-485-3p has been previously identified as a pathway and therapeutic target in neuroinflammatory disease, and the potential relevance of this target in PD was further confirmed through RNA-Seq and analysis of expression levels in animal models and patients with PD.

Neurology/Psychiatric

A novel CAPN2-targeting ASO, AMX-0114, rescues neurite degeneration in vitro

March 24, 2023

No Comments

Calcium-dependent protease calpain-2 (CAPN2) has been previously proposed as a critical effector of axonal degeneration, which is a key early...

Neurology/Psychiatric

Quralis announces program targeting UNC13A mis-splicing for neurodegenerative diseases

March 20, 2023

No Comments

Quralis Corp. has launched a program that targets UNC13A mis-splicing, a critical genetic alteration...

Businesswoman pressing dollar sign on touchscreen

Quralis closes $88M series B round for trials of targeted ALS therapies

March 9, 2023

By Cormac Sheridan

No Comments

Quralis Inc. raised $88 million in series B round to fund clinical development of its two lead programs in amyotrophic lateral sclerosis (ALS) and to take forward earlier-stage pipeline projects in ALS and frontotemporal dementia.

A microscopic image of liver tissue affected by metabolic dysfunction-associated steatotic liver disease.

Endocrine/Metabolic

Seed funding at Resalis to support development of ncRNA-based therapeutics for metabolic disease

Feb. 23, 2023

No Comments

Resalis Therapeutics Srl has completed a seed financing round of €10 million (US$10.6 million) to establish a noncoding RNA (ncRNA)-based...

Newco news

Resalis closes $11M seed round to develop miR inhibitor for metabolic disease

Feb. 22, 2023

By Cormac Sheridan

No Comments

Resalis Therapeutics Srl closed a €10 million (US$10.6 million) seed round to progress toward the clinic a micro-RNA (miR) inhibitor in development for metabolic disease indications. Riccardo Panella, chief scientific officer and founder, identified the potential of a particular miR species, miR-22, as a target for metabolic syndrome while conducting studies on its potential role in oncology while at Harvard Medical School and Beth Israel Deaconess Medical Center.

Drug Design, Drug Delivery & Technologies

Vanda and Olipass collaborate to develop antisense oligonucleotide therapeutics

Sep. 30, 2022

No Comments

Vanda Pharmaceuticals Inc. and Olipass Corp. have entered into a research and development collaboration agreement to jointly develop a set of antisense oligonucleotide (ASO) molecules based on Olipass' proprietary modified peptide nucleic acids.

Tofersen turnaround: Lead investigator sees new upsides in phase III Biogen ALS trial

Sep. 21, 2022

By Nuala Moran

No Comments

It failed to meet the primary endpoint at six months, but the European chief investigator for Biogen Inc.’s phase III trial of tofersen in treating amyotrophic lateral sclerosis (ALS) now describes the study as “trailblazing,” following a six-month open label extension.

Articles Tagged with ''antisense oligonucleotides''

Vanda's ASO VCA-894A awarded US orphan drug designation for Charcot-Marie-Tooth disease type 2S

Stoke Therapeutics’ ASO STK-002 cleared to enter clinic in U.K. for autosomal dominant optic atrophy

BMD-001, a nanoparticle-formulated ASO with efficacy in preclinical models of Parkinson’s disease

A novel CAPN2-targeting ASO, AMX-0114, rescues neurite degeneration in vitro

Quralis announces program targeting UNC13A mis-splicing for neurodegenerative diseases

Quralis closes $88M series B round for trials of targeted ALS therapies

Seed funding at Resalis to support development of ncRNA-based therapeutics for metabolic disease

Resalis closes $11M seed round to develop miR inhibitor for metabolic disease

Vanda and Olipass collaborate to develop antisense oligonucleotide therapeutics

Tofersen turnaround: Lead investigator sees new upsides in phase III Biogen ALS trial

Today's news in brief

Today's news in brief

Boryung invests $10M in Intuitive Machines for outer space R&D

Phase II Huntington’s tie-up with Novartis could edge $3B for PTC

Elevar nabs Relay’s FGFR2 rare cancer drug in $500M deal