CSL Behring

DPHARM 2024 focused on innovations in clinical trials, featuring keynotes on ROI, DCTs, AI, and patient perspectives. Panels discussed efficiency, AI use, and ecosystem approaches. Outside-industry speakers offered insights on decision-making and innovation mindset. Technologists and sponsors highlighted patient-centric services and strategic collaborations. DPHARM Idol Disrupt showcased new technologies, with Power winning for a patient-matching platform. The event aimed to improve trial efficiency and deliver life-changing medicines.

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.

Graft versus Host Disease Market to grow at 9.9% CAGR (2020-2034), driven by branded therapies, strong pipeline, and increased awareness. Market size in the US was $1 billion in 2023, expected to grow at 7.4% CAGR (2024-2034). Emerging therapies like Axatilimab and CSL964 to transform market. Key companies include Equillium, Biocon, Takeda, and CSL Behring.

The graft versus host disease market is growing due to the adoption of branded therapies like JAKAFI, IMBRUVICA, ORENCIA, and REZUROCK, a strong pipeline with over 10 new drugs expected to launch, and increased awareness of GvHD management. Innovative treatments and ongoing research aim to address unmet needs in this complex condition.

DPHARM 2024 panel discussed adapting pharma operating models for efficient clinical trials, emphasizing the need to address scientific, technological, and regulatory complexities. Medicare's drug price negotiations may lower physician reimbursement. California's new 'deepfake' laws aim to regulate AI-generated content in elections. Deloitte found 50% of US women skip or delay medical care due to cost, access, or negative experiences, impacting long-term health and wealth. Parexel's poll revealed nearly 40% see the need for more agile service models in clinical trials, aligning with the rise of Functional Service Provider (FSP) models.

DelveInsight's 'Hemophilia B Pipeline Insight, 2024' report details the global Hemophilia B treatment market, including clinical trials, therapies, and company developments. Key companies and therapies in various stages of development are highlighted, along with market drivers and barriers.

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.