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FDA approves Sarclisa combination for multiple myeloma

Drug Discovery World

It is approved in combination with bortezomib, lenalidomide, and dexamethasone for adults with who are not eligible for autologous stem cell transplant (ASCT). The efficacy of the treatment was evaluated in the IMROZ trial in patients with newly diagnosed multiple myeloma who were not eligible for ASCT.

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Actigen moves closer to clinical trial for rare disease treatment  

Drug Discovery World

Biotech company Actigen has moved closer to clinical trials for rare disease treatment for Hunter syndrome, following a pre-Investigational New Drug (IND) meeting with the US Food and Drug Administration (FDA). ? . Hunter syndrome .

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International Women’s Day: Female life science leaders

Drug Discovery World

For International Women’s Day, DDW’s Diana Spencer celebrates some of the women in leadership making an impact on the drug discovery and life sciences sector. She has managed multinational projects for charities and not-for-profits to find new treatments for Malaria and led Innovate UK grants through to successful completion.

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Potential treatment for patients with acute myeloid leukaemia 

Drug Discovery World

Overcoming Resistance to FLT3 Inhibitors in the Treatment of FLT3-Mutated AML. International Journal of Molecular Science, 21(4): 1537. Around one third of patients with AML are diagnosed with FLT3-mutations, which are associated with a higher risk of relapse and poor clinical outcome. and Leung, A. and Wang, H-G.

Treatment 130
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Patients show 91% response to allogeneic CAR-T therapy

Drug Discovery World

Investigators saw a 91% overall response rate (ORR) in patients with relapsed/refractory multiple myeloma (RRMM) following treatment with allogeneic CAR-T therapy P-BCMA-ALLO1 and lymphodepletion. P-BCMA-ALLO1 is an investigational non-viral, stem cell memory T cell (TSCM)-rich allogeneic CAR-T cell therapy.

Therapies 147
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First therapy for very rare blood clotting disorder gets US greenlight

Drug Discovery World

Takeda’s Adzynma has become the first recombinant protein product approved in the US for prophylactic or on-demand enzyme replacement therapy (ERT) for congenital thrombotic thrombocytopenic purpura (cTTP). For prophylactic ERT, Adyznma is administered to help reduce the risk of disease symptoms.

Therapies 130
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Bispecific antibody successfully reduced bleeds in haemophilia A

Drug Discovery World

The trial assessed both once-weekly and once-monthly prophylactic treatment with the investigational treatment Mim8. The data were presented at the International Society of Thrombosis and Haemostasis Annual Congress (ISTH 2024) in Bangkok, Thailand. for those who received no prior prophylaxis treatment.