Breaking news in research

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  • Santhera announces collaboration to develop CMD gene therapy 

    21 May 2019

    Santhera Pharmaceuticals announced today that it has started a collaboration with the Biozentrum, University of Basel, to develop a gene therapy for LAMA2-deficient congenital muscular dystrophy (CMD). The potential gene therapy has shown positive results in mouse models of the condition and researchers will now seek to explore the feasibility of delivering it using a virus. Santhera is also developing a drug for CMD called omigapil, which could be a complementary treatment to the gene therapy.

    For more information, read Santhera’s press release.

  • Update on Solid’s DMD gene therapy trial

    14 May 2019

    Solid Biosciences has provided an update on its IGNITE-DMD trial, building on the preliminary results released back in February. Two new boys have been included in the study. One of them has received a higher dose of the gene therapy SGT-001, while the other one has been assigned to the control group. In a blood test that measures liver function, the boy that was dosed had abnormal results, and this was considered to be a serious side effect. However, the patient quickly responded to an increased dose of oral steroids and is now doing well. IGNITE DMD is ongoing and continues to be open for enrolment.

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

  • Scholar Rock announces the start of its Phase II SMA trial

    09 May 2019

    Scholar Rock has announced today that it has begun dosing patients in its Phase II clinical trial in Type 2 and Type 3 SMA. The trial is testing SRK-015, a drug that aims to build muscle mass by blocking a protein called myostatin. Scholar Rock is hoping to enrol approximately 55 patients across the U.S., Canada, and Europe.

    For more information read Scholar Rock’s press release.

  • Sarepta acquires rights to sixth LGMD gene therapy

    08 May 2019

    Sarepta Therapeutics announced today that it has signed an agreement with the Research Institute at Nationwide Children’s Hospital (Ohio, USA) to develop a gene therapy for LGMD type 2A. This will be the sixth LGMD gene therapy that the company is developing, with three of them already in clinical trials. This new gene therapy aims to replace the CAPN3 gene, which is faulty in people with LGMD 2A.

    For more information read Sarepta’s press release.

  • Positive data announced for three SMA therapies

    07 May 2019

    The 71st annual meeting of the American Academy of Neurology, held this week, has seen the release of positive data from multiple SMA clinical trials. This includes additional data on the already approved Spinraza, as well as new positive data on two potential therapies under development: Zolgensma and risdiplam.

    Read Biogen’s press release on Spinraza.

    Read AveXis’ press release on Zolgensma.

    Read Roche’s press release on risdiplam

  • More positive data from MTM gene therapy trial

    02 May 2019

    Audentes Therapeutics has announced new positive data from its Phase 1/2 clinical trial in X-linked myotubular myopathy. 11 patients are taking part in the study and results so far show improvements in muscle and respiratory function. The majority of children treated with the gene therapy have been able to gradually achieve milestones such as head control, sitting unassisted, crawling and standing with support. Ventilator use has also been reduced or even stopped.

    For more information, read Audentes’ press release.

  • Clinical trial for centronuclear myopathy given the green light in the UK

    30 April 2019

    The drug company Dynacure has announced today that UK regulators have approved its UNITE-CNM phase I/II clinical trial. UNITE-CNM will be a first-in-human study of the drug candidate DYN101, and will enrol patients greater than 16 years of age with X-linked and autosomal dominant centronuclear myopathy. It is expected to begin in the second half of 2019.

    For more information, read Dynacure’s press release.

  • Fulcrum repurpose drug for FSHD

    24 April 2019

    Fulcrum Therapeutics has obtained rights to losmapimod from GlaxoSmithKline (GSK). This drug has been tested in 24 clinical trials for multiple indications, including heart and lung disease, so it is known to be generally safe. Fulcrum is interested in losmapimod because it has found that the drug can switch off DUX4 in cells originating from people with facioscapulohumeral muscular dystrophy (FSHD). The company plans to initiate a phase 2b trial testing losmapimod in people with FSHD at clinical sites in the US and Europe in mid 2019.

    For more information read Fulcrum’s press release.

  • Positive preliminary results from SMA gene therapy trial

    17 April 2019

    AveXis has shared preliminary data from its phase 3 clinical trial (STR1VE) in spinal muscular atrophy type 1. The treatment, Zolgensma, is a gene therapy designed to replace the faulty gene that causes the condition. The trial results so far show that children treated with Zolgensma are living longer and achieving significant development milestones.

    For more information read AveXis’ press release.

  • Wave announces details of its upcoming DMD trial

    16 April 2019

    Today, Wave Life Sciences shared details of its upcoming Phase II/III clinical trial testing suvodirsen in boys with DMD. Suvodirsen is an exon-skipping drug that targets exon 51 within the dystrophin gene. The study, named DYSTANCE 51, will start in July 2019 and enrol boys who are between 5 and 12 years of age. Participants will receive suvodirsen or placebo once a week for 48 weeks.

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

  • Audentes starts therapy development for DMD and myotonic dystrophy

    8 April 2019

    Today, Audentes Therapeutics has announced that it will begin the development of new genetic medicines for Duchenne muscular dystrophy and myotonic dystrophy type 1. These therapies will aim to address the underlying causes of these conditions by delivering genetic information into the body. Audentes currently has an AAV-based gene therapy for myotubular myopathy in early-stage clinical trial.

    For more information read Audentes’ community letter and press release.

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