Other rare inherited disorders

Snijders Blok-Campeau syndrome, (NCT06860672)
Sponsors:
Yongguo Yu

IND Enabling

Phase I

Phase II

Phase III

Refractory pain due to small fiber neuropathy (iSFN), (NCT06980948)
Sponsors:
Sangamo Therapeutics

IND Enabling

Phase I

Phase II

Phase III

Alpha 1-Antitrypsin Deficiency, AATD, (NCT06677307)
Sponsors:
Korro Bio

IND Enabling

Phase I

Phase II

Phase III

Alpha-1 Antitrypsin Deficiency, AATD, (NCT06622668)
Sponsors:
Intellia Therapeutics

IND Enabling

Phase I

Phase II

Phase III

MECP2 Duplication Syndrome, MDS, (NCT06615206)
Sponsors:
HuidaGene Therapeutics Co., Ltd.

IND Enabling

Phase I

Phase II

Phase III

Alpha-1 Antitrypsin Deficiency, AATD, (NCT06405633)
Sponsors:
Wave Life Sciences Ltd.

IND Enabling

Phase I

Phase II

Phase III

Alpha 1-Antitrypsin Deficiency, AATD, (NCT06389877)
Sponsors:
Beam Therapeutics Inc.

IND Enabling

Phase I

Phase II

Phase III

Alpha-1 Antitrypsin Deficiency, AATD, (NCT06186492)
Sponsors:
Wave Life Sciences Ltd.

IND Enabling

Phase I

Phase II

Phase III

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