AskBio leverages its Pro10 AAV manufacturing process that is used by several pharma giants. The company holds several patents in areas such as AAV production, chimeric vectors, and self-complementary DNA. The company’s technology has already proved its worth by winning regulatory approval for gene therapy developed for Spinal muscular atrophy by Illinois-based AveXis, a subsidiary of Novartis. Pfizer is also leveraging AskBio’s proprietary gene-delivery platform for developing novel therapies for patients with Hepatitis C virus (HCV).
Novartis’ Iptacopan (LNP) Shows Promise in C3 Glomerulopathy (C3G)
Novartis announced positive interim Phase II results of its most advanced kidney drug, Iptacopan (LNP) in C3 glomerulopathy (C3G) at the virtual American Society of Nephrology (ASN) 2020 Annual Meeting. As per estimates presented by NORD, C3G affects persons 2–3 per 1,000,000 people, thus an ultra-rare and severe type of primary glomerulonephritis.
Iptacopan is a first-in-class oral, small molecule that inhibits factor B, which is a key serine protease of the alternative pathway of the complement cascade. The drug is simultaneously under investigation for a number of renal conditions associated with complement system dysregulation, including IgA nephropathy, paroxysmal nocturnal hemoglobinuria (PNH), atypical hemolytic uremic syndrome, and membranous nephropathy.
The drug also received Orphan drug designation a few months ago, won the PRIME designation in C3 glomerulopathy (C3G) that will offer the drug advantage to the patients.
Forma Therapeutics’ Olutasidenib Proves Efficient in IDH1-Mutated AML
Forma Therapeutics’ lead candidate, Olutasidenib, has demonstrated clinically efficient and safe activity in Phase II FT2102-HEM-101 clinical trial in patients with relapsed/refractory (R/R) acute myeloid leukemia (AML) with mutations in isocitrate dehydrogenase 1 (IDH1m). The company acknowledges that IDH1 mutations occur in 6% to 8% of all the AML patients, in nearly 80% of brain cancer cases including grade 2 and 3 gliomas and secondary glioblastoma.
Olutasidenib is a selective inhibitor of IDH1m, an enzyme, which is responsible for causing blood cancers and solid tumors when mutated. The trial is not only examining olutasidenib as monotherapy in IDH1m AML but in combination with azacitidine or cytarabine in cohorts in patients with IDH1m AML or myelodysplastic syndrome (MDS).
EpicGenetics Enters a Research Collaboration with Mayo Clinic to Bolster Fibromyalgia Diagnostics
EpicGenetics, a privately held biomedical company based in Los Angeles, announced a contract with the Mayo Clinic Fibromyalgia and Chronic Fatigue Clinic to advance research to better diagnose fibromyalgia. The duo plans to investigate the use of the EpicGenetics FM/a Test to determine the test’s use and applicability for the same.
Fibromyalgia (FM) is a common rheumatological syndrome characterized by chronic, diffuse musculoskeletal pain, and tenderness with a number of associated symptoms, among which sleep disturbances, fatigue, and affective dysfunction affecting over 20 million patients in the US alone.
