Researchers working at the Lady Davis Institute, Jewish General Hospital, McGill University, have combined whole-exome sequencing (WES) from nearly 300,000 samples from the multiancestry UK Biobank, with ultrasound-derived heel estimated bone mineral density (eBMD) and genome-wide association (GWAS) data to identify potential future therapeutic targets for patients with osteoporosis.
Abcuro Inc. pulled down an oversubscribed $155 million series B financing co-led by Redmile Group and Bain Capital Life Sciences to advance cytotoxic T and natural killer cells therapies. Specifically, proceeds will back the phase II/III registrational trial of ABC-008, a first-in-class anti-killer cell lectin-like receptor G1 (KLRG1) antibody for inclusion body myositis (IBM) as well as fund continued development of other clinical programs.
There are plenty of companies chasing the first U.S. FDA-approved treatment for the ultra-rare disease fibrodysplasia ossificans progressiva (FOP), but Ipsen SA stayed the course longer and won that approval Aug. 16 with Sohonos (palovarotene). Other companies have struggled to develop a treatment for FOP, which affects about 400 people in the U.S. and about 900 people worldwide. Some potential competitors are still in the clinic, trying to catch up.
Cgenetech (Suzhou, China) Co. Ltd. has divulged cyclopropanamide-containing compounds acting as Janus kinase (JAK) inhibitors, particularly JAK2 and/or JAK1 and/or JAK3, reported to be useful for the treatment of rheumatoid arthritis, ulcerative colitis, atopic dermatitis, systemic lupus erythematosus, essential thrombocythemia, polycythemia vera and myelofibrosis.
The U.S. FDA has given its first approval for treating fibrodysplasia ossificans progressiva (FOP), an ultra-rare disease that creates bone formation outside the skeleton that can lead to immobility, life-threatening respiratory problems and a total locking of the jaw.
Modalis Therapeutics Corp. – previously Edigene Corp. – has regained full rights to two gene therapy candidates for muscle disorders, MDL-201 and MDL-202, that were co-developed with Astellas Pharmaceutical Inc. since 2019.
Variable muscle expression of frataxin (FXN) protein in muscle plus five adverse events (AEs) of injection-site thrombophlebitis in the multiple ascending-dose (MAD) phase I study hamstrung Design Therapeutics Inc. in its Friedreich’s ataxia (FA) bid with DT-216. Wall Street punished the Carlsbad, Calif.-based firm’s stock (NASDAQ:DSGN) with a knockdown of 70%, or $5.17, leaving shares at $2.17 when the market closed Aug. 15.
The U.S. FDA has approved Daxxify (daxibotulinumtoxinA-lanm) for treating cervical dystonia in adults nearly a week ahead of its Aug. 19 PDUFA date. Approval of the sBLA for the injectable, peptide-formulated neuromodulator went to Revance Therapeutics Inc. The drug’s original approval was for treating glabellar lines, better known as frown lines, came in September 2022. Daxxify is the first neuromodulator stabilized with Revance’s peptide exchange technology. It is created without human serum albumin or animal-based components.
Modalis Therapeutics Corp. – previously Edigene Corp. – has regained full rights to two gene therapy candidates for muscle disorders, MDL-201 and MDL-202, that were co-developed with Astellas Pharmaceutical Inc. since 2019.
Diabetes has been associated with increased bone fracture risks, however, the cellular and molecular mechanisms underlying diabetic bone fragility are not clear. In the current study, researchers from the Children's Hospital of Philadelphia presented data from a study that aimed to investigate these mechanisms in murine monogenic model of type 1 diabetes (T1D).
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