sickle cell anemia
Beam Therapeutics Reports Chemo-Related Patient Death in Trial of Base-Editing Sickle Cell Drug
Investigators determined that the patient died due to respiratory failure likely related to busulfan but unrelated to BEAM-101.
Editas Seeking Partner or Licensing Deal for Lead Sickle Cell, TDT Gene-Editing Treatment Candidate
The strategic shift will allow the firm to focus on advancing a newly announced in vivo gene-editing therapy candidate that's in preclinical development.
During a panel discussion at the National Society of Genetic Counselors meeting, genetic counselors discussed the risks and benefits of gene therapies.
Vertex Grabs Rights to Orum's Targeted Protein Degradation Technology
The deal, valued at more than $325 million, gives Vertex the right to use Orum's technology to develop new targeted conditioning agents for its gene therapies.
Vertex Pharmaceuticals Sues HHS, OIG Over Legality of Fertility Preservation Program
The firm is asking a court to determine that its program to pay for fertility treatments for patients on Casgevy doesn't violate anti-kickback and inducement laws.