Breaking news in research

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  • Solid Biosciences report on DMD gene therapy trial

    7 February 2019

    Solid Biosciences today announced preliminary findings from its IGNITE-DMD trial, which has so far tested SGT-001 in three boys. Muscle biopsies taken after three months showed low levels of micro-dystrophin protein. Although this is disappointing news, it’s important to remember that IGNITE-DMD is a dose-escalation study, and these three boys received the lowest planned dose. These results confirm that a higher dose of SGT-001 will need to be tested in future patients.

    For further information, please read Solid’s community letter and press release.

  • CMT drug receives Fast Track Designation

    5 February 2019

    French pharmaceutical company Pharnext has announced that PXT3003 has been granted Fast Track Designation by the US Food and Drug Administration (FDA). PXT3003 is a combination of three already-approved drugs that could potentially treat Charcot Marie Tooth disease type 1A (CMT 1A). Fast Track Designation gives Pharnext certain benefits that will help with the development of PXT3003.

    For further information, please read Pharnext’s press release.

  • Wave selected for innovative clinical trial programme

    4 January 2019

    Wave Lifesciences’ next Duchenne trial has been accepted onto a new FDA programme to advance and promote innovative trial designs. The company plans to make use of historical control data in order to reduce the number of patients in the placebo arm of its phase 2/3 trial. We welcome this news and hope that the FDA programme leads to lots of shared learnings for the rare disease field.

    For further information, please read Wave’s press release.

  • LGMD 2D gene therapy receives Orphan Drug Designation

    3 January 2019

    Myonexus Therapeutics’ gene therapy for limb girdle muscular dystrophy type 2D, MYO-102, has been granted Orphan Drug Designation by the US Food and Drug Administration (FDA). This gives Myonexus certain benefits that will help with the development of MYO-102.

    For further information, please read Myonexus’ press release.

  • Roche’s SMA drug receives priority designation

    18 December 2018

    Roche’s drug for spinal muscular atrophy, risdiplam (RG7916), has been accepted onto PRIME. This is a scheme run by the European Medicines Agency (EMA) to enhance support for the development of medicines that target an unmet need. Obtaining PRIME status gives Roche various benefits, including early dialogue and scientific advice from EMA.

    For further information, please read Roche’s press release.

  • Positive results from Wave’s DMD trial

    7 December 2018

    Wave Life Sciences has announced positive results from its phase 1 clinical trial testing exon 51 skipping drug WVE-210201. The trial showed that the drug was safe and well-tolerated. Based on these results, Wave has selected a dose for its phase 2/3 trial, which should start in 2019.

    For further information, please read Wave’s press release

  • FDA fast tracks SMA gene therapy

    4 December 2018

    Novartis has announced that the US Food and Drug Administration (FDA) has accepted its marketing application for AVXS-101 for the treatment of spinal muscular atrophy (SMA) type 1. The gene therapy has been granted a Priority Review by the FDA, which is a quicker review process than normal. The FDA’s decision is expected to be announced in May 2019.

    For further information, read Novartis’ press release

  • CMT drug receives fast track designation

    28 November 2018

    Acceleron Pharma has announced the US Food and Drug Administration has granted Fast Track Designation to ACE-083, for the treatment of individuals with Charcot-Marie-Tooth Disease (CMT). This status will help speed up the development and regulatory review process of the drug.

    ACE-083 is currently being evaluated in a two-part Phase 2 trial. Preliminary results from the second part of the trial are expected by the end of 2019.

    For more information, please read Acceleron’s press release.

  • Update on Solid’s DMD gene therapy trial

    14 November 2018

    Solid Biosciences has announced two additional boys have been dosed in its gene therapy trial – IGNITE DMD. Six boys have now been enrolled into IGNITE DMD in the USA; three have been treated and are currently doing well. The remaining three boys are part of a delayed-treatment control group.

    Solid is continuing to enrol boys into IGNITE DMD and expects to release preliminary trial results in the first quarter of 2019.

    For further information, please read Solid Biosciences’ press release.

  • Update on FSHD drug development programme

    01 November 2018

    Facio Therapies has shown that one of its lead compounds can reduce the levels of DUX4 in a mouse model. The company now plans to do further testing of its lead candidates before it can move to a clinical trial in 2021.

    For more information, please read Facio Therapies’ press release.

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