Thousands of Californians live with sickle cell disease, a painful blood disorder. But new treatments, including a promising form of gene therapy, are offering hope to ...
A one-time gene-editing therapy, exagamglogene autotemcel (exa-cel), has demonstrated sustained and clinically meaningful ...
Pociredir Phase 1b PIONEER Trial Data in Sickle Cell Disease to be Presented and Published, Including 12 mg and 20 mg Cohorts ―― Live and ...
Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company developing precision genetic medicines through base editing, today announced that the company will present updated safety and efficacy ...
Life expectancy has soared and interest in the disease is growing, but breakthrough gene therapies are extraordinarily ...
Azim, MD, MS, Division Chief Transplant and Cell Therapy/Hematological malignancies at Loma Linda University (LLU) Cancer ...
Details regarding the oral presentation can be found below. Abstract Title: Accelerating Access to Gene Therapy: Lessons from Commercial Implementation in Sickle Cell Disease and Transfusion-Dependent ...
CorrectSequence Therapeutics' CS-121 Completed Dosing of First Chylomicronemia Patient, Demonstrating Excellent Safety and ...
Detailed price information for Vertex Pharmaceutic (VRTX-Q) from The Globe and Mail including charting and trades.
VRTX's new drugs deliver a mixed Q3 performance. Alyftrek sales surge. Journavx and Casgevy lag expectations but show promise.
Understanding how cells turn genes on and off is one of biology's most enduring mysteries. Now, a new technology developed by chemist Brian Liau and his collaborators at Harvard offers an ...
For kids with the condition, day-to-day activities like going to school or playing with friends can lead to life-threatening ...
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