. Author manuscript; available in PMC: 2024 Aug 31.

Published in final edited form as: N Engl J Med. 2023 Aug 31;389(9):820–832. doi: 10.1056/NEJMoa2215643

Table 1.

Participant Demographics and Outcomes.

	Participant 1	Participant 2	Participant 3
Demographics and disease severity
Age (at screening)/sex	22 years/male	21 years/male	24 years/female
Sickle cell disease genotype	β^S/β^S	β^S/β^S	β^S/β^S
Sickle cell disease - related symptoms before enrollment	Six episodes of acute chest syndrome over the past 10 years and a history of a silent cerebral infarct, retinopathy, and priapism.	Four vaso-occlusive episodes, 3 episodes of acute chest syndrome, and a silent cerebral infarct during the preceding 20 years.	Twenty five vaso-occlusive pain episodes in the 2 years prior to enrollment.
Sickle cell disease treatment ongoing at study enrollment	Chronic blood transfusions and hydroxyurea.	Hydroxyurea.	Chronic blood transfusions and hydroxyurea.
Apheresis collection and OTQ923 manufacture
Mobilization cycles (Lasting 2–3 d each)	3	2	3
Cell dose manufactured, ×10⁶ cells/kg	2.8, a combination of 2 manufacturing batches, each with 84% editing efficiency	5.99, a combination of 3 batches with editing efficiencies of 78%, 75%, and 73%, respectively	5.04, a combination of 2 batches with editing efficiencies of 87% and 82%, respectively
Follow-up and outcomes
Neutrophil engraftment	Day +26	Day +20	Day +18
Platelet engraftment	Day +44	Day +29	Day +29
AEs experienced since OTQ923 infusion	36	16	45
AEs related to OTQ923	0	0	0
Follow-up since OTQ923 infusion	18 months	12 months	6 months
Sickle cell disease - related complications since OTQ923 infusion^†	One vaso-occlusive crisis episode with acute chest syndrome occurring at 17 months after infusion. Recurrent intermittent priapism. No new stroke, or silent cerebral infarct. Continued mild hemolysis. Worsening osteonecrosis of femur.	One vaso-occlusive crisis episode occurring at 12 months after infusion. No acute chest syndrome, stroke or priapism. Continued mild hemolysis. Persistent osteonecrosis of femoral head.	One vaso-occlusive crisis occurring at 9 months after infusion.^* Continued mild hemolysis. Persistent osteonecrosis of femoral head.

^†

The observation period for post-treatment sickle cell–related events starts on the day of first OTQ923 infusion and ends on the day of last follow-up.

AEs, adverse events.

This event happened after the data cut off and hence the rest of the follow up is only up to 6 months.