News & Events

News January 8, 2021

Alpha Thalassemia Major: No Longer a Fatal Disease

Virtual Conference on Alpha Thalassemia Major

January 8, 2021

Prenatal exome sequencing identifies genetic causes of non-immune hydrops fetalis

October 8, 2020

BrainVar catalogues genetic variants that underlie brain development – prenatally, postnatally, through childhood

Photo of Green Glia Cells

Fetal molecular therapies show promise in studies of MPS7 mice

Administering stem cell or enzyme therapy in utero may be a path to alleviating some congenital diseases that often result […]

Photo of Tippi MacKenzie, MD, working

Fixing nature’s genetic mistakes in the womb

On November 18, 2019, we convened an In Utero Gene Therapy Think Tank in San Francisco to expand the discussion […]

December 11, 2019

Scientist working in lab

Time Magazine covers clinical trial for alpha thalassemia major

Our Co-Founder, Tippi MacKenzie, MD, was featured in an article in Time Magazine discussing the alpha thalassemia major (ATM) clinical […]

September 18, 2018

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