Upcoming Cell & Gene Therapy PDUFA’s in 2024

Several highly anticipated PDUFA decisions are approaching..👇

Abeona Therapeutics ( $Abeona Therapeutics(ABEO.US)$ )
Asset: Pz-cel (prademagene zamikeracel)
PDUFA Date: May 25, 2024
Indication: Recessive dystrophic epidermolysis bullosa (RDEB)
Technology: Autologous cell therapy, treatment uses gene transfer to deliver COL7A1 genes into an RDEB patient’s own skin cells then transplanting them back to the patient.

Rocket Pharmaceuticals ( $Rocket Pharmaceuticals(RCKT.US)$ )
Asset: Marnetegragene autotemcel RP-L201 (KRESLADI™)
PDUFA Date: June 30, 2024
Indication: Immunodeficiency, Leukocyte Adhesion Deficiency
Technology: Gene therapy that contains autologous hematopoietic stem cells that have been genetically modified with a lentiviral vector to deliver a functional copy of the ITGB2 gene.

Adaptimmune Therapeutics ( $Adaptimmune Therapeutics(ADAP.US)$ )
Asset: Afami-cel (Afamitresgene autoleucel) (ADP-A2M4)
PDUFA Date: August 4, 2024
Indication: Advanced synovial sarcoma
Technology: Engineered T-cell receptor (TCR) T-cell therapy targeted to the MAGE A4 cancer target.

Autolus Therapeutics ( $Autolus Therapeutics(AUTL.US)$ )
Asset: Obecabtagene autoleucel (obe-cel)
PDUFA Date: November 16, 2024
Indication: Relapsed/refractory (r/r) Adult B-Cell Acute Lymphoblastic Leukemia (ALL)
Technology: Autologous CD19 CAR T cell therapy with a unique CD19 CAR.