. 2024 Dec 18;12:156. doi: 10.1186/s40364-024-00701-x

Table 4.

Active clinical trials testing therapeutic approaches based on the CRISPR/Cas12 or Cas13 systems [139]

NCT Number	Study Title	Study Status	Study Overview	Sponsor	Phase
NCT04853576	A Study Evaluating the Safety and Efficacy of EDIT-301 in Participants With Severe Sickle Cell Disease (RUBY)	RECRUITING	Multicenter study to evaluate the safety and efficacy of a single dose of EDIT-301 in subjects with severe sickle cell disease. Treatment: EDIT-301 (autologous gene edited (CD)34⁺ hematopoietic stem cells, by Cas12a) administered as a one-time intravenous infusion, after myeloablative conditioning with busulfan. Outcome measures: − Proportion of subjects achieving complete resolution of severe vaso-occlusive events. − Frequency and severity of adverse events.	Editas Medicine	Phase 1/2
NCT05444894	EDIT-301 for Autologous Hematopoietic Stem Cell Transplant (HSCT) in Participants With Transfusion-Dependent Beta Thalassemia (TDT)	RECRUITING	Single-arm, multicenter study evaluating the safety, tolerability, and efficacy of a single unit dose of EDIT-301 in adult participants with TDT, age 18 to 35 years. Treatment: EDIT-301 (autologous gene edited (CD)34⁺ hematopoietic stem cells, by Cas12a) administered as a one-time intravenous infusion, after myeloablative conditioning with busulfan. Outcome measures: − Proportion of participants achieving engraftment. Frequency and severity of adverse events.	Editas Medicine, Cambridge, MA, USA	Phase 1/2
NCT06031727	CRISPR/Cas13-mediated RNA tarGeting tHerapy for the Treatment of Neovascular Age-related Macular Degeneration Investigator-initiated Trial (SIGHT-I)	RECRUITING	Multicenter trial to evaluate the safety, tolerability, and efficacy of CRISPR/Cas13 RNA-editing therapy HG202, targeting knock-down of Vascular Endothelial Growth Factor A (VEGFA), in the treatment of neovascular Age-related Macular Degeneration (nAMD). Treatment: Once unilateral subretinal injection of HG202, a CRISPR/Cas13 RNA-editing therapy, which use one single vector to partially knock-down the expression of VEGFA and thus inhibit choroidal neovascularization formation in patients who are either responsive or non-responsive to anti-VEGF agents. Outcome measures: Incidence and severity of ocular and systemic adverse events.	HuidaGene Therapeutics, Shanghai, PRC	Phase 1

NCT Number

Study Title

Study Status

Study Overview

Sponsor

Phase

NCT04853576

A Study Evaluating the Safety and Efficacy of EDIT-301 in Participants With Severe Sickle Cell Disease (RUBY)

RECRUITING

Multicenter study to evaluate the safety and efficacy of a single dose of EDIT-301 in subjects with severe sickle cell disease.

Treatment: EDIT-301 (autologous gene edited (CD)34⁺ hematopoietic stem cells, by Cas12a) administered as a one-time intravenous infusion, after myeloablative conditioning with busulfan.

Outcome measures:

− Proportion of subjects achieving complete resolution of severe vaso-occlusive events.

− Frequency and severity of adverse events.

Editas Medicine

Phase 1/2

NCT05444894

EDIT-301 for Autologous Hematopoietic Stem Cell Transplant (HSCT) in Participants With Transfusion-Dependent Beta Thalassemia (TDT)

RECRUITING

Single-arm, multicenter study evaluating the safety, tolerability, and efficacy of a single unit dose of EDIT-301 in adult participants with TDT, age 18 to 35 years.

Treatment: EDIT-301 (autologous gene edited (CD)34⁺ hematopoietic stem cells, by Cas12a) administered as a one-time intravenous infusion, after myeloablative conditioning with busulfan.

Outcome measures:

− Proportion of participants achieving engraftment.

Frequency and severity of adverse events.

Editas Medicine, Cambridge, MA, USA

Phase 1/2

NCT06031727

CRISPR/Cas13-mediated RNA tarGeting tHerapy for the Treatment of Neovascular Age-related Macular Degeneration Investigator-initiated Trial (SIGHT-I)

RECRUITING

Multicenter trial to evaluate the safety, tolerability, and efficacy of CRISPR/Cas13 RNA-editing therapy HG202, targeting knock-down of Vascular Endothelial Growth Factor A (VEGFA), in the treatment of neovascular Age-related Macular Degeneration (nAMD).

Treatment: Once unilateral subretinal injection of HG202, a CRISPR/Cas13 RNA-editing therapy, which use one single vector to partially knock-down the expression of VEGFA and thus inhibit choroidal neovascularization formation in patients who are either responsive or non-responsive to anti-VEGF agents.

Outcome measures: Incidence and severity of ocular and systemic adverse events.

HuidaGene Therapeutics, Shanghai, PRC

Phase 1