AATD may affect 100,000 Americans. And it’s generally driven by a single misspelled letter, one that can be corrected with Prime’s technology. advertisement.

Shares in Wave Life Sciences rose by 74% after it reported clinical data with a pioneering new RNA-editing therapy for alpha-1 antitrypsin deficiency (AATD). Wave claims this is the first clinical ...

Bernstein has upgraded Beam Therapeutics (NASDAQ:BEAM) to outperform from market perform, citing upcoming data on the company’s drug candidate for alpha-1 antitrypsin deficiency, or AATD. The ...

Korro Bio recently began a Phase 1/2 test of KRRO-110, an RNA-editing therapy for AATD. Last summer, biotech startup AIRNA raised $60 million to advance to the clinic in 2025 with its experimental ...

Wave Life Sciences' stock soars on positive initial data showing the first-ever therapeutic RNA editing in humans in the phase Ib/IIa study of WVE-006 for AATD.

CAMBRIDGE, Mass., March 10, 2025 (GLOBE NEWSWIRE) -- Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company developing precision genetic medicines through base editing, today announced ...

--Beam Therapeutics Inc., a biotechnology company developing precision genetic medicines through base editing, today announced the first patient was treated with BEAM-302, an investigational in ...

Alpha-1 antitrypsin deficiency (AATD) is a genetic disorder associated with liver disease, ranging from fibrosis to hepatocellular carcinoma. The disease remains asymptomatic until its final ...

Vertex said phase 1 biomarker analyses showed its two AAT correctors “would not deliver transformative efficacy for people with AATD.” Unable to go big, the biotech decided to go home ...