Rare Disease

| CRISPR Medicine | This is an overview of articles and trials related to Rare Diseases.

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Disease Roundup: Genomic Medicine Candidates for...

Jul. 20, 2022

This up-to-date roundup presents the 7 clinical-stage gene-editing approaches to sickle cell disease, which affects millions of people worldwide and...

Clinical Update: First Child Dosed in CRISPR Trial...

Apr. 13, 2022

Editas Medicine announced earlier this week that it had dosed the first child in the BRILLIANCE clinical trial of EDIT-101 for Leber congenital...

Clinical Update: CRISPR Therapy for Transthyretin...

Mar. 2, 2022

In this week's update, we provide news about Intellia Therapeutics' and Regeneron's ongoing Phase 1 trial for NTLA-2001 in transthyretin amyloidosis....

CRISPR Clinical Trial Roundup: Rare Diseases

Feb. 16, 2022

In this week's clinical trial update, we summarise the ongoing in vivo CRISPR clinical trials for three distinct rare genetic diseases: hereditary...

CRISPR Medicine in 2021 – What a Phenomenal Year...

Dec. 28, 2021

In 2021, we've seen positive clinical data for the first ever in vivo CRISPR medicines, base editors are heading for the clinic, and CRISPR therapies...

Safer, More Efficient AAV Systems for Therapeutic...

Dec. 20, 2021

A recent proof-of-concept study from the Sontheimer lab at the University of Massachusetts Chan Medical School uses all-in-one, self-inactivating AAV...

CRISPR Clinical Trials – Latest News Updates

Dec. 1, 2021

This week’s CRISPR trial update brings you the latest developments from ongoing CRISPR clinical trials, including expansion of Intellia Therapeutics’...

Webinar

Rare Diseases & CRISPR

Nov. 17, 2021

Read more about the webinar - Rare Diseases & CRISPR

Rare Disease

| CRISPR Medicine | This is an overview of articles and trials related to Rare Diseases.

Sign-up to weekly Newsletter here.

Hereditary Angioedema, HAE (NCT05120830)
Sponsors:
Intellia Therapeutics

IND Enabling

Phase I

Phase II

Phase III

Sickle Cell Disease, SCD, (2018-001320-19)
Sponsors:
Vertex Pharmaceuticals Incorporated

IND Enabling

Phase I

Phase II

Phase III

Sickle Cell Disease, SCD, (NCT04774536)
Sponsors:
Principal Investigator: Mark Walters, MD UCSF Benioff Children's Hospital Oakland

IND Enabling

Phase I

Phase II

Phase III

Beta-Thalassemia, BT, (NCT04205435)
Sponsors:
Bioray Laboratories - Bangyao Biotechnology Co., Ltd.

IND Enabling

Phase I

Phase II

Phase III

Sickle Cell Disease, SCD, (NCT04819841)
Sponsors:
Graphite Bio Inc

IND Enabling

Phase I

Phase II

Phase III

Sickle Cell Disease, SCD, (NCT04443907)
Sponsors:
Novartis Pharmaceuticals

IND Enabling

Phase I

Phase II

Phase III

Beta-Thalassemia, BT, (NCT03728322)
Sponsors:
Allife Medical Science and Technology Co., Ltd

IND Enabling

Phase I

Phase II

Phase III

Herpes Simplex Virus Refractory Keratitis, (NCT04560790)
Sponsors:
Shanghai BDgene Co., Ltd.

IND Enabling

Phase I

Phase II

Phase III