Tag: FSHD
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…
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,…
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…
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…
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 …
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…
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…
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…
