Albatroz Therapeutics Pte Ltd. has secured $3 million in funding to accelerate the development of therapeutic antibodies against a novel target that degrades the extracellular matrix, a key contributor to cancer and arthritis.
A twisted, uncertain path has led to a U.S. FDA approval for Biogen Inc.’s Qalsody (tofersen), the first drug targeting a genetic cause of amyotrophic lateral sclerosis (ALS). Qalsody is for ALS associated with a mutation in the superoxide dismutase 1 (SOD1) gene. Patients with SOD-1 mutations account for 2% of ALS cases.
Dupuytren’s disease is often referred to as “the most common crippling hand condition that people have never heard of,” but Kurt Harrington is on a mission to change that. A seasoned biotech and pharma consultant, Harrington has had the condition himself for over a decade and, acutely aware of the lack of available treatment options, has founded Ventoux Biosciences Inc. to bring additional therapies forward.
Janssen Pharmaceutica NV has disclosed IL-17A/IL-17 receptor A (IL-17RA) interaction modulators reported to be useful for the treatment of psoriasis, rheumatoid arthritis, radiographic axial spondyloarthritis (ankylosing spondylitis), multiple sclerosis, asthma and more.
Bone remodeling involves a process of continuously cycling bone formation by osteoblasts balanced by resorption by osteoclasts. Development of osteoclast cells is initiated by RANKL and macrophage colony-stimulating factor (M-CSF) ligands leading to differentiation of bone marrow-derived monocytes (BMDMs) to generate osteoclast specified cells expressing TRAP, CTSK and MMP9.
Bone development is a continuous process, but in some cases, soft tissues can mineralize due to some anomalies in repairing processes, thus leading to heterotopic ossification (HO). Max Planck Institute for Molecular Genetics researchers aimed to find the genetic causes tied to this abnormal bone-formation disorder.
Abbvie Inc. recently disclosed the discovery and structure of the anti-tumor necrosis factor (TNF) glucocorticoid receptor modulator (GRM) immunology antibody-drug conjugate (iADC) ABBV-154. The drug is in phase II clinical development as a subcutaneous treatment for rheumatoid arthritis and polymyalgia rheumatica, and as a subcutaneous or intravenous treatment for active Crohn’s disease.
The editing in human cells and in mice of the survival motor neuron 1 gene (SMN1) restored the levels of SMN protein that the mutation of the SMN2 gene produces in spinal muscular atrophy. Scientists from the Broad Institute in Boston and The Ohio State University reversed the mutation using the base editing technique.
The editing in human cells and in mice of the survival motor neuron 1 gene (SMN1) restored the levels of SMN protein that the mutation of the SMN2 gene produces in spinal muscular atrophy (SMA). Scientists from the Broad Institute in Boston and The Ohio State University reversed the mutation using the base editing technique. “This base editing approach to treating SMA should be applicable to all SMA patients, regardless of the specific mutation that caused their SMN1 loss,” the lead author David Liu, a professor and director of the Merkin Institute of Transformative Technologies in Healthcare at the Broad Institute of Harvard and MIT, told BioWorld.
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