The new case of myelodysplastic syndrome (MDS) that overshadowed Bluebird Bio Inc.’s earnings and resulted in a clinical hold by the FDA will be addressed similarly to an earlier hitch in the sickle cell disease program, said Andrew Obenshain, the company’s head of severe genetic diseases. “Essentially, we will try and do the same thing,” and exonerate the lentiviral vector, he said during a conference call with investors. “At this point, we don't have tumor cells or leukemia to analyze,” he noted.
Sinocelltech Group Ltd. won market approval from China’s NMPA for SCT-800, a B-domain deleted recombinant human coagulation factor VIII, for the prophylactic treatment of severe hemophilia A in adolescent and adult patients. This marks the first homegrown drug for treating hemophilia A in China.
LONDON – Hemab ApS has closed a $55 million series A to take forward treatments for bleeding and thrombosis disorders based on antibodies in-licensed from Novo Nordisk A/S, where the newco’s founder, Johan Henrik Faber, previously led hemophilia drug research.
Officials at Sigilon Therapeutics Inc. declined to comment beyond a press release on the FDA’s clinical hold for the phase I/II study with encapsulated cell therapy SIG-001 for severe or moderately severe hemophilia A. Shares of Cambridge, Mass.-based Sigilon (NASDAQ:SGTX) closed at $6.90 on July 9, down $2.34, or 25% after Wall Street learned of the regulatory move, which came because one of three patients treated has developed inhibitors to factor VIII (FVIII).
Gene therapy specialist Uniqure N.V. said Tuesday that, with confidence inspired by new 52-week data on its investigational hemophilia B therapy, etranacogene dezaparvovec, it plans to submit a BLA for the program with partner CSL Behring LLC in first quarter of 2022. Uniqure meanwhile has moved to acquire Corlieve Therapeutics SAS and its lead program to treat temporal lobe epilepsy, the most common form of focal epilepsy. The acquisition, worth up to €250 million (US$297.3 million) for Corlieve, includes €46.3 million cash up front.
New and updated preclinical and clinical data presented by biopharma firms at the European Hematology Association 2021 Virtual Congress, including: Celltrion, Crispr, Daiichi Sankyo, Debiopharm, Janssen, Kite, Omeros, TG, Vertex, X4.
Beigene Ltd. unveiled positive interim results from its Alpine phase III trial comparing its small-molecule BTK inhibitor, Brukinsa (zanubrutinib), to Abbvie Inc.’s Imbruvica (ibrutinib), lending validity to one of the company’s most important development programs.
New and updated preclinical and clinical data presented by biopharma firms at the European Hematology Association 2021 Virtual Congress, including: Abbvie, Acceleron, Agios, Alexion, Aptose, Autolus, Beigene, Bergenbio, Bluebird, BMS, Caelum, Celyad, Constellation, Curis, Editas, Equillium, Forma, Genentech, Global Blood, Imago, Imara, Immunicum, Keros, Menarini, Mustang, Novartis, Oryzon, Protagonist, Ryvu.
The FDA has lifted clinical holds on four studies from Bluebird Bio Inc., following recent similar actions with other gene therapy programs. Two of the studies concern phase I/II and phase III clinical trials of the gene therapy Lentiglobin (BB-1111) in treating sickle cell disease. The remaining two studies are phase III clinical trials of betibeglogene autotemcel gene therapy, which share a vector with Lentiglobin, for treating transfusion-dependent beta-thalassemia.
Now that Apellis Pharmaceuticals Inc.’s Empaveli pegcetacoplan has won FDA approval as the first targeted C3 therapy for treating paroxysmal nocturnal hemoglobinuria (PNH), the cost of treating the rare blood disorder is hefty.
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