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BEAM-101 Receives RMAT for the Treatment of Sickle Cell Disease

Pharmacy Times

Completion Date (Estimated): February 1, 2027 BEAM-101 is an investigational, genetically modified cell therapy that consists of autologous CD34+ hematopoietic stem and progenitor cells that have been base-edited in the promoter regions of the HBG1/2 genes. Worldwide, about 8 million people have the disease.

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Taletrectinib Gains FDA Approval for Locally Advanced or Metastatic ROS1+ NSCLC

Pharmacy Times

Completion Date (Estimated): June 2027 “For people living with advanced ROS1+ lung cancer, who tend to be diagnosed at a younger age, having another treatment option can make a real difference for them and their loved ones,” Janet Freeman-Daily, cofounder and president of The ROS1ders, said in a news release.

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Sarepta to lay off about 500 employees after Duchenne gene therapy setbacks

BioPharma Dive

Sarepta is working on a new regimen of immune-suppressing drugs to manage the safety risks, and plans to soon share its findings with the FDA. The company also has $1 billion in senior notes coming due in 2027. The company believes sales in ambulatory patients are likely to annualize at a minimum of $500 million annually through 2027.

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Moving towards oral delivery of biologics

European Pharmaceutical Review

1 Used to treat a range of chronic diseases (eg, diabetes, rheumatoid arthritis, psoriasis, Crohn’s disease, haemophilia, etc), it is projected that the sales of biologics will rise from $380 billion in 2022 to $416 billion in 2023, and to almost $600 billion in 2027.

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Covid-19 vaccine success bolsters nanoparticle drug delivery research

Pharmaceutical Technology

With early nanoparticle studies, researchers often found the human body’s innate immune response to drugs and the short-lasting effects of drugs challenging, as they can also reduce a drug’s efficacy. Since then, the drug has been used off-label in breast cancer and other types of cancer.