One of the issues with gene therapy using AAV vectors is that patients develop neutralizing antibodies against the vector, which may keep the patient from receiving a second dose of the gene therapy treatment, if needed.
Sangamo Therapeutics has announced that they are ready to start the first-ever trial of gene editing in the human body with a study of gene therapy for hemophilia B.
On May 31, 2017, FDA approved Rebinyn, Novo Nordisk’s longer-acting GlycoPEGylated recombinant factor IX product.
Dimension Therapeutics has decided to end their DTX101 development program for hemophilia B gene therapy.
CSL Behring has received the National Organization for Rare Disorders (NORD) 2017 Industry Innovation Award for Idelvion, their longer-acting factor IX product.
Catalyst Biosciences has received IND approval from South Korea for a Phase I/II clinical study of their subcutaneous recombinant factor IX candidate CB 2679d/ISU304.
Bioverativ has reported results from their ongoing Phase III trial of Alprolix in children under 12.
Aptevo Therapeutics has announced that data from its ongoing Phase III clinical trial of Ixinity recombinant factor IX shows that the product is safe and effective in children under 12.