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 ...

AIRNA, a biotech company pioneering RNA editing therapeutics to restore the health of patients with rare and common diseases, today announced the appointment of Matthew Hawryluk, Ph.D., MBA, as Chief ...

Morgan Stanley downgraded Intellia Therapeutics (NTLA) to Equal Weight from Overweight with a price target of $11, down from $56. The firm now ...

which is in Phase 1 clinical trials for treating Alpha-1 antitrypsin deficiency; and KB301 that is in Phase 2 clinical trials for treating aesthetic skin conditions, as well as in open label study ...

Intellia Therapeutics, Inc. shares declined over 5% on Monday morning after Morgan Stanley downgraded the stock to ‘Equal ...

The discontinued program, dubbed NTLA-3001, targets a rare lung disease known as alpha-1 antitrypsin deficiency. Intellia had begun a Phase 1/2 clinical trial testing the therapy in August.

Liver diseases can be inherited (such as Wilson disease and alpha 1-antitrypsin deficiency) or acquired (for example hepatitis and liver cancer). The performance of AAV gene-therapy vectors is ...

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.

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 ...

of its total workforce and is stopping work on its investigational med NTLA-3001 in alpha-1 antitrypsin deficiency-associated lung disease as well as “select research-stage programs.” ...

Baird downgraded Leap Therapeutics (LPTX) to Neutral from Outperform with a price target of $1.25, down from $9, after the ...