CRISPR Therapeutics, Inc.

Pfizer withdraws Oxbryta® due to 16 patient deaths in clinical trials, citing imbalance in vaso-occlusive crises and fatal events. The European Medicines Agency reports 18 deaths in two Phase III trials, with 16 linked to the drug. Pfizer halts all clinical trials and expanded access programs globally.

Pfizer withdraws Oxbryta, its sickle cell disease treatment, from global markets due to increased risks of vaso-occlusive crises and fatal events. The decision follows recent FDA approvals of two gene therapies for SCD, Casgevy and Lyfgenia, which offer potential cures but come with high costs.

DelveInsight's 'Renal Cancer Pipeline Insight 2024' report highlights a robust pipeline with 70+ companies developing 75+ therapies, including key players like Genentech and Merck. Promising drugs like Atezolizumab and ADI-270 are under various clinical trial phases, with recent FDA Fast Track designations for ADI-270 in metastatic RCC.

DelveInsight's 'Renal Cancer Pipeline Insight 2024' report highlights a robust pipeline with 70+ companies developing 75+ therapies, including key players like Genentech, Allogene Therapeutics, and Merck & Co. Inc. Promising therapies such as Atezolizumab and ALLO-316 are under various clinical trial phases, with recent FDA Fast Track designations and IND approvals for novel drugs like ADI-270 and AB-2100.

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Infusions of Vertex's Casgevy and bluebird bio's Lyfgenia for sickle cell treatment have begun, marking a breakthrough. Both companies face a slow uptake due to the lengthy treatment process, but interest is high. Vertex aims to activate 75 centers globally, while bluebird expects a consistent revenue stream from Lyfgenia.

The Global Gene Therapy Market is valued at USD 2.4 billion in 2024, growing at a CAGR of 19.25% during 2024-2035. Gene therapies aim to treat genetic disorders by introducing healthy genes or inactivating faulty ones. The market has seen significant advancements, with 345 companies involved in developing therapies, and 1,100 active clinical trials in April 2024. By 2034, gene therapies are expected to treat over 65.6 million patients.

Bluebird Bio's $3.1 million gene therapy Lyfgenia for sickle cell disease has seen slow uptake, with only four patients treated since its launch. Payer approval and patient readiness are cited as significant barriers. The high cost and complex reimbursement process highlight systemic issues in the adoption of cell and gene therapies. Despite slow initial uptake, experts anticipate a gradual increase as the healthcare system adapts to transformative treatments.

Gene editing therapeutics market estimated at $11 million in 2024, forecasted to grow at 147% CAGR to reach $1 billion by 2029, driven by chronic diseases, rare disorders, strategic initiatives, and precision medicine.

CRISPR-Cas9 gene editing therapy Casgevy, approved for sickle cell disease, faces slow patient treatment and profitability challenges. 20 patients are 'in the funnel' but timeline for treatment remains unclear. Insurance coverage for Casgevy's $2.2 million price is uncertain, with commercial payers likely to cover first. Fertility treatments not covered by Medicare/Medicaid add complexity. Future gene therapies need streamlined manufacturing and robust clinical data for market success.