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Exon skipping news

You are here: Home / News / Exon skipping news

PepGen Announces First Patient Dosed in CONNECT1-EDO51 Phase 2 Clinical Trial of PGN-EDO51 for Duchenne Muscular Dystrophy Patients Amenable to Exon 51 Skipping

January 11, 2024 by John Marrin

PepGen Inc., a clinical-stage biotechnology company advancing the next-generation of oligonucleotide therapies, today announced that the first patient …

PepGen Announces First Patient Dosed in CONNECT1-EDO51 Phase 2 Clinical Trial of PGN-EDO51 for Duchenne Muscular Dystrophy Patients Amenable to Exon 51 SkippingRead More

Wave Life Sciences Announce the Start of the FORWARD-53 Clinical Study 

December 21, 2023 by John Marrin

Wave Life Sciences Announce the Start of the FORWARD-53 Clinical Study  On the 15th of December 2023, Wave Life Sciences announced the initiation …

<strong>Wave Life Sciences Announce the Start of the FORWARD-53 Clinical Study </strong>Read More

PepGen Announces Clearance by Health Canada of CTA for PGN-EDO51 to Begin the Phase 2 Clinical Trial, CONNECT1-EDO51, for the Treatment of Duchenne Muscular Dystrophy

May 19, 2023 by Mehreen Arif

PepGen Announces Clearance by Health Canada of CTA for PGN-EDO51 to Begin the Phase 2 Clinical Trial, CONNECT1-EDO51, for the Treatment of Duchenne …

PepGen Announces Clearance by Health Canada of CTA for PGN-EDO51 to Begin the Phase 2 Clinical Trial, CONNECT1-EDO51, for the Treatment of Duchenne Muscular DystrophyRead More

Dyne Therapeutics Receives FDA Orphan Drug and Rare Pediatric Designations for DYNE-251 for the Treatment of Duchenne Muscular Dystrophy

March 25, 2023 by Lizzie Cox

Dyne Therapeutics Receives FDA Orphan Drug and Rare Pediatric Designations for DYNE-251 for the Treatment of Duchenne Muscular Dystrophy Dyne …

Dyne Therapeutics Receives FDA Orphan Drug and Rare Pediatric Designations for DYNE-251 for the Treatment of Duchenne Muscular DystrophyRead More

Nationwide researchers announce restoration of full-length Dystrophin in humans

May 27, 2022 by Mehreen Arif

In Duchenne muscular dystrophy, dystrophin protein is absent or partially functional due to mutations in the dystrophin gene. Multiple therapeutic …

Nationwide researchers announce restoration of full-length Dystrophin in humansRead More

Dyne Therapeutics announce application to start exon 51 skipping clinical trial

December 7, 2021 by Lynnette

Dyne Therapeutics, Inc. announced on 2nd December 2021 the submission of an Investigational New Drug (IND) application to the U.S. Food and Drug …

Dyne Therapeutics announce application to start exon 51 skipping clinical trialRead More

Project that aimed to combine stem cell and CRISPR technology is a success

October 28, 2021 by Neil

We’re delighted to report the successful completion of our research project in Dr Yung-Yao Lin’s laboratory at Queen Mary University of London. The …

Project that aimed to combine stem cell and CRISPR technology is a successRead More

In just 7 weeks, we’ll be on the road again

May 4, 2021 by Samantha

Duchenne Science on Tour 2 Where we'll be helping our families to make informed choices around really complex things like gene therapy and exon …

In just 7 weeks, we’ll be on the road againRead More

Golodirsen gains conditional approval in US

December 16, 2019 by Neil

Sarepta Therapeutics has announced that the FDA (the medicines regular in the US) has granted accelerated approval to golodirsen.  Golodirsen, or …

Golodirsen gains conditional approval in USRead More

Wave halts development of exon skipping drugs

December 16, 2019 by Neil

Wave has today announced that they have discontinued the clinical program of suvodirsen (designed to skip exon 51 of the dystrophin gene) and are …

Wave halts development of exon skipping drugsRead More

Duchenne Science events back on the road in 2020

December 5, 2019 by Lynnette

We've welcomed over 100 people to our Science on Tour events throughout 2019, these empowering and invaluable sessions have been a huge success and we …

Duchenne Science events back on the road in 2020Read More

Wave gives suvodirsen update

September 19, 2019 by Neil

Wave Life Sciences announced earlier this week that Suvodirsen has been granted fast track approval by the Federal Drug Administration (the drug …

Wave gives suvodirsen updateRead More

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