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268,233 Results for "exonics therapeutics".
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Duchenne muscular dystrophy
Dyne’s Duchenne Exon Skipping Oligomer Shows ‘Differentiated’ Clinical Effect
Dyne is eyeing an accelerated approval filing for DYNE-251 in early 2026 that would pit the asset against Sarepta’s Exondys 51 in a patient population amenable to exon 51 skipping.
March 17, 2025
·
2 min read
·
Tristan Manalac
Duchenne muscular dystrophy
Wave’s Duchenne Exon-Skipper Reverses Muscle Damage in Mid-Stage Trial
WVE-N531, an oligonucleotide, elicited significant functional benefit and reversal of muscle damage in the Phase II FORWARD-53 trial. Wave plans to file for accelerated approval of the candidate in 2026.
March 26, 2025
·
3 min read
·
Heather McKenzie
Business
Ascidian Therapeutics Enters Collaboration with Roche for Discovery and Development of RNA Exon Editing Therapeutics Targeting Neurological Diseases
Ascidian Therapeutics today announced a research collaboration and licensing agreement with Roche (SIX: RO, ROG; OTCQX: RHHBY) for the discovery and development of RNA exon editing therapeutics targeting neurological diseases.
June 18, 2024
·
3 min read
Press Releases
Entrada Therapeutics Receives Authorization in the United Kingdom to Initiate ELEVATE-45-201, a Phase 1/2 Multiple Ascending Dose Clinical Study of ENTR-601-45 in People Living with Duchenne Muscular Dystrophy Amenable to Exon 45 Skipping
March 25, 2025
·
7 min read
Business
Exonate Appoints Dr Rafiq Hasan as Non-Executive Director
Exonate Ltd., a biotechnology company developing novel, non-invasive, small-molecule therapeutics for patients with retinal vascular diseases, announced the expansion of its Board with the appointment of Dr Rafiq Hasan as Non-Executive Director.
May 28, 2024
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2 min read
Business
Roche Inks Potential $1.8B Deal With Ascidian to Develop RNA Exon Therapies
Ascidian Therapeutics will receive $42 million upfront from Roche, and up to $1.8 billion in milestone payments and royalties, to discover RNA exon editing candidates for neurological diseases.
June 18, 2024
·
2 min read
·
Tyler Patchen
Press Releases
Taiho Pharmaceutical, Taiho Oncology, and Cullinan Therapeutics Announce Primary Endpoint Met in Phase 2b Trial of Zipalertinib in Patients with Non-Small Cell Lung Cancer Harboring EGFR Exon 20 Insertion Mutations Who Have Received Prior Therapy
January 28, 2025
·
8 min read
Press Releases
HUTCHMED Announces NMPA Full Approval for ORPATHYS® (savolitinib) in China for Patients with Locally Advanced or Metastatic MET Exon 14 NSCLC
January 13, 2025
·
11 min read
Press Releases
ORIC Pharmaceuticals Announces Clinical Supply Agreement to Evaluate ORIC-114 in Combination with Amivantamab for the First-Line Treatment of NSCLC with EGFR Exon 20 Insertion Mutations
January 14, 2025
·
6 min read
Genetown
Ascidian Therapeutics to Present Preclinical Data for RNA Exon Editor, ACDN-01, Supporting an Open IND for Phase 1/2 Clinical Testing at ASGCT 2024 Annual Meeting
Ascidian Therapeutics, a biotechnology company seeking to treat human diseases by rewriting RNA, today announced that it will share preclinical data that enabled IND clearance for Phase 1/2 testing for its lead RNA exon editing therapy, ACDN-01, in a poster session at the American Society of Gene & Cell Therapy (ASGCT) annual meeting in Baltimore, MD, May 7-11, 2024.
April 25, 2024
·
3 min read
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