. 2023 Oct 20;31:101125. doi: 10.1016/j.omtm.2023.101125

Table 4.

Other Cell and Gene Therapies clinical holds

Sponsor/Program/Trial identifier #	Target disease	Dates of hold	Reason for hold	Status/Conclusions
Verve Therapeutics⁶⁴ VERVE-101 (Non-viral gene editing treatment) N/A	Hetero-zygous familial hyperchol-esterolemia (HeFH)	Nov-22 – Ongoing as of Dec-2022	FDA requests additional preclinical data relating to: (1) potency differences between human and non-human cells, (2) risks of germline editing, and (3) off-target analyses in non-hepatocyte cell types. The FDA also requested available clinical data from the ongoing heart-1 trial. In addition, the FDA has requested modification of the trial protocol in the United States to incorporate additional contraceptive measures and to increase the length of the staggering interval between dosing of participants.	Verve intends to submit a response as expeditiously as possible. No clinical trial number at this time.
Gamida Cell⁶⁵^,⁶⁶ GDA-201 (Cryopreserved NK Cell therapy in combination with rituximab.) NCT05296525	Follicular and diffuse large B cell lymphomas.	Oct-21 – Apr-22	FDA had requested modifications in donor eligibility procedures and sterility assay qualification (CMC).	Trial was put on hold after IND application and before initiation of dosing.
Vertex⁶⁷^,⁶⁸ VX-880 (Stem cell-derived, fully differentiated pancreatic islet cell replacement therapy) NCT04786262	Type 1 diabetes	May-22 – July-22	Clinical hold was a “surprise” to sponsor. To date, there have been no serious adverse events, and the first two patients treated with half the target dose established proof of concept.	FDA determined that there was not enough evidence to support increasing the treatment dose as planned. So far, evidence supporting product benefit in patients is strong. No serious adverse events.
Editas Medicine⁶⁹^,⁷⁰ EDIT-301 (HSCT edited cell therapy) NCT04853576	Sickle cell disease	Jan-21 – July-22	Partial clinical hold. Company passed safety phase but was required to develop and submit an improved potency assay prior to enrolling the efficacy phase.	Improved potency assay.