Tag: FSHD

Epic Bio Reports New Data on Hypercompact Cas Proteins

– Data being presented at ESGCT reflect improvements upon CasMINI, previously the smallest Cas protein known to work in human cells – – Company’s additional poster presentations report on progress of epigenetic editing platform and lead candidate EPI-321 – SOUTH SAN FRANCISCO, Calif., Oct. 26, 2023 (GLOBE NEWSWIRE) — Epic…

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Enrollment complete in Phase 3 trial of losmapimod for FSHD

Fulcrum Therapeutics has completed patient enrollment for its Phase 3 clinical trial evaluating losmapimod as a potential treatment for people with Facioscapulohumeral muscular dystrophy (FSHD). The study, dubbed REACH (NCT05397470), is evaluating the efficacy and safety of losmapimod against a placebo in FSHD adult patients across 33 sites in the…

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The FSHD Society Celebrates Fulcrum’s Milestone in the Fight Against FSHD

Fulcrum Therapeutics has announced successful completion of patient enrollment in REACH Phase 3 trial RANDOLPH, Mass., Sept. 8, 2023 /PRNewswire-PRWeb/ — The FSHD Society, a leading advocate and partner in the quest to find treatments for Facioscapulohumeral Muscular Dystrophy (FSHD), is thrilled to announce a significant milestone in the battle against…

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Fulcrum Announces Completion of Enrollment in the Phase 3

CAMBRIDGE, Mass., Sept. 07, 2023 (GLOBE NEWSWIRE) — Fulcrum Therapeutics, Inc.® (Nasdaq: FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today announced that it has completed enrollment in REACH, a Phase 3 clinical trial evaluating losmapimod in patients with FSHD at…

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Six biotech spinouts you should watch out for this year

Academic spinouts are companies that are typically formed from research taking place at academic institutions and are sometimes launched and owned by the university. A means to turn research into products of commercial value, the biotech industry has witnessed quite a few spinouts develop from research projects over the years….

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SMCHD1 and LRIF1 converge at the FSHD-associated D4Z4 repeat and LRIF1 promoter yet display different modes of action

Somatic loss of LRIF1 or SMCHD1 in control myocytes leads to insufficient DUX4 derepression We have previously shown that SMCHD1 and LRIF1L aid in transcriptional repression of the D4Z4 repeat as short-term depletion of either LRIF1L or SMCHD1 in muscle cells having a D4Z4 repeat of <20 units on a…

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Kate Therapeutics launches to develop genetic medicines

Gene therapy company Kate Therapeutics Inc. (KateTx), has emerged from stealth mode with a $51 million Series A financing. In addition, the company has granted Astellas Pharma Inc. an exclusive, worldwide license to develop, manufacture and commercialize KT430 to treat X-linked myotubular myopathy (XLMTM). There are a large number of…

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Celularity Tissue Factor Gene Knockout of Allogeneic

Data published online in the journal Cytotherapy demonstrate that CRISPR-mediated tissue factor gene knockout (TFKO) in allogeneic stromal cells (ASCs) leads to significantly lower tissue factor (TF) expression, activity, and thrombotic effects, providing a feasible strategy potentially to improve the safety of ASC-based cell therapy FLORHAM PARK, N.J., June 01,…

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Celularity Tissue Factor Gene Knockout of Allogeneic Stromal Cells Significantly Lowers Thrombotic Effects; Study Highlights Critical Importance of Gene Editing Capability

Celularity Inc. (Nasdaq: CELU) (“Celularity”), a biotechnology company developing placental-derived allogeneic cell therapies and biomaterial products, announced today the online publication of preclinical data in Cytotherapy, the official journal of the International Society of Cell and Gene Therapy (ISCT). The paper, “Characterization of CRISPR/Cas9-edited human placental allogenic stromal cells with…

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Fulcrum Therapeutics: FSHD Steadily Advancing In Phase 3 (NASDAQ:FULC)

24K-Production Shares of small molecule developer Fulcrum Therapeutics (NASDAQ:FULC) have lost more than 80% of their value since the IPO was priced at $19 in August 2021. Year to date performance is negative 50% after FTX-6058 for sickle cell disease was placed on full clinical hold by the FDA in…

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Avidity Biosciences Announces FDA Eases Partial Clinical Hold on AOC 1001 Providing a Clear Path Forward to Finalize Pivotal Dose and Phase 3 Design in Adults with Myotonic Dystrophy Type 1

  Avidity plans to double the number of participants receiving 4 mg/kg of AOC 1001 in MARINA Open-Label Extension (MARINA-OLE™) study by dose escalating participants currently on 2 mg/kg of AOC 1001 FDA allows new participant enrollment for 2 mg/kg of AOC 1001 …

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Toxic protein linked to both muscular dystrophy and arhinia

National Institutes of Health (NIH) researchers and collaborators have found that DUX4, a toxic protein made by the body, may be responsible for two very different rare genetic disorders. The study, published on Friday in Science Advances, may eventually lead to therapies that can help people with a type of…

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Utilisation of exome sequencing for muscular disorders in Thai paediatric patients: diagnostic yield and mutational spectrum

We performed ES in 43 Thai paediatric patients with MD or CM from 42 unrelated families. Their clinical features and WES data were analysed simultaneously, enabling the correlation of the ES results with other diagnostic modalities. The overall diagnostic yield for ES was 70% (30/43). The most common MD-disease genes…

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Human microRNA inhibits expression of pathogenic gene underlying facioscapulohumeral muscular dystrophy

Fig. 1: miR-675 targets the DUX4 ORF and 3′UTR. a RenLuc-DUX4-FL dual-luciferase reporter and CMV.H19 constructs, and U6.MIR675, H1.MIR675, U6.MIR675-3p, and U6.MIR675-5p miRNAs. RenLuc-DUX4-FL contains DUX4 ORF and 3′UTR sequences fused to Renilla luciferase after the stop codon. Exons 1–3 are indicated (Ex1,2,3). *SD5 indicates silent mutation of a cryptic…

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