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CAR-T therapy for nasopharyngeal cancer enters Phase II trials

Drug Discovery World

The US Food and Drug Administration (FDA) has approved a Phase II clinical trial for Biosyngen’s BRG01, an EBV-specific CAR-T cell therapy. This marks the first cell therapy to enter Phase lI trials in both the US and China for the treatment of relapsed/metastatic EBV-positive nasopharyngeal carcinoma.

Therapies 130
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Great Britain first to approve CRISPR-based gene therapy

Drug Discovery World

The Medicines and Healthcare products Regulatory Agency (MHRA) has authorised the world’s first gene therapy for sickle-cell disease (SCD) and transfusion-dependent β-thalassemia (TDT). Of these eligible patients, 28 (97%) were free of severe pain crises for at least 12 months after treatment.

Therapies 277
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CHMP okays first therapy targeting FGFR3 alterations in bladder cancer

Drug Discovery World

It is recommended as a monotherapy to treat adult patients with unresectable or metastatic urothelial carcinoma, harbouring susceptible FGFR3 genetic alterations who have previously received at least one line of therapy containing a PD-1 or PD-L1 inhibitor in the unresectable or metastatic treatment setting. months vs 7.8 months vs 2.7

Therapies 147
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How to advance AAV-based gene therapies

Drug Discovery World

Sara Donnelly, Director of Research Planning and Business Development at PhoenixBio USA explores why the right pre-clinical model is essential for teams wanting to advance adeno-associated virus vector-based gene therapies. Adeno-associated virus (AAV) vector-based gene therapies hold exceptional promise across a range of disease areas.

Therapies 162
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Janssen submits EMA filing for bladder cancer treatment 

Drug Discovery World

Patients with mUC, including FGFR-driven tumours, face a particularly poor prognosis and the need for innovative therapies remains high. Patients with mUC, including FGFR-driven tumours, face a particularly poor prognosis and the need for innovative therapies remains high.

Treatment 130
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EU orphan designation for pulmonary arterial hypertension drug

Drug Discovery World

The drug was approved by the FDA for Expanded Access, allowing trial participants to continue CS1 treatment when no comparable or satisfactory alternative therapy options are available. Topline results will be shared in Q3, 2024.

Drugs 264
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‘Ice Bucket Challenge’ funds FDA-approved ALS treatment

Drug Discovery World

The US Food and Drug Administration (FDA) has granted approval to AMX0035, a new treatment for people living with amyotrophic lateral sclerosis (ALS). million from the ALS Association, raised by the 2014 social media craze the ‘Ice Bucket Challenge’ Another 40 potential new treatments are being investigated. .