Beam Therapeutics shows promise in gene therapy for sickle cell disease, but it also has high risks and market expectations.

Alpha-1 antitrypsin deficiency (AATD) is a genetic disorder that leads to low levels of a protein called alpha-1 antitrypsin (AAT) in the blood. This deficiency can result in serious health issues ...

Wave Life Sciences develops stereopure oligonucleotides targeting genetic mutations, with lead assets in DMD, AATD, and Huntington's Disease showing promising data. Major collaborations with GSK ...

(Getty Images) A mysterious illness plagued Kristin Hatcher’s family for 4 generations, until her father was diagnosed with AATD. WASHINGTON—At 42 years old, Kristin Hatcher has the sad distinction of ...

Beam Therapeutics Inc. (NASDAQ:BEAM), a biotechnology company at the forefront of precision genetic medicines with a market capitalization of $1.87 billion, is navigating a critical juncture in its ...

Current PROMs used in AATD reflect the COPD aspect of the disease but fail to address the extra-pulmonary issues these patients face. A more discriminative and comprehensive patient-reported outcome ...

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 ...

In 2025, we expect to continue this momentum as we advance our differentiated, high-impact clinical programs in DMD, HD and AATD and bring our innovative therapeutic approach for obesity into the ...

for the treatment of alpha-1 antitrypsin deficiency (AATD) associated lung disease. Also, with the strategic reorganization, NTLA is looking to reduce its current workforce by almost 27% over the ...

Beam Therapeutics BEAM is developing its lead pipeline candidate, BEAM-101, a base-editing therapy, in the phase I/II BEACON study for treating sickle cell disease (SCD), an inherited blood disorder.

Keep an eye on the first patient dosed in phase 1 and 2 trials of BEAM-302 in Alpha-1 Antitrypsin Deficiency (AATD),with initial clinical data expected in 2025.Plus,new players in the biotech ...