Immunodeficiencies

Researchers have engineered probiotic bacteria to produce CRISPR-Cas9 and edit the ROS-generating NOX2/gp91phox target in mouse intestinal cells. The...

AEGIS Targets Personalised Editing for Childhood...

CMN Briefs

Jul. 31, 2026

An IGI-led consortium has received up to $27.7 million from ARPA-H to develop personalised in vivo gene-editing treatments for children with inborn...

Gene Replacement Emerges as New Immunodeficiency...

CMN Interview

May. 27, 2024

Two research groups are independently pioneering a groundbreaking gene-editing treatment for severe immunodeficiencies, potentially transforming the...

Immunodeficiencies

X-Linked Severe Combined Immunodeficiency, (NCT06851767)
Sponsors:
National Institute of Allergy and Infectious Diseases (NIAID)

IND Enabling

Phase I

Phase II

Phase III

CD40L-HyperIgM Syndrome, (NCT06959771)
Sponsors:
National Institute of Allergy and Infectious Diseases (NIAID)

IND Enabling

Phase I

Phase II

Phase III

Chronic Granulomatous Disease, (NCT06559176)
Sponsors:
Prime Medicine

IND Enabling

Phase I

Phase II

Phase III

Hereditary Angioedema, HAE, (NCT06634420)
Sponsors:
Intellia Therapeutics

IND Enabling

Phase I

Phase II

Phase III

Chronic Granulomatous Disease, CGD, (NCT06325709)
Sponsors:
National Institute of Allergy and Infectious Diseases (NIAID)

IND Enabling

Phase I

Phase II

Phase III

Hereditary Angioedema, HAE, (NCT05120830)
Sponsors:
Intellia Therapeutics

IND Enabling

Phase I

Phase II

Phase III