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Beam Therapeutics

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Prime Medicine's PM647 has received FDA clearance to enter first-in-human testing as the company advances a one-time Prime Editing therapy targeting the genetic cause of alpha-1 antitrypsin deficiency. Representative image.
Pharma & Biotech

Prime Medicine stock jumps after FDA clears PM647 gene-editing trial for alpha-1 antitrypsin deficiency

Prime Medicine shares jumped in after-hours trading after the FDA cleared PM647 for first-in-human testing, moving a one-time gene-editing therapy for alpha-1 antitrypsin deficiency from promising animal data into the much harder test of clinical development.

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Beam Therapeutics has begun dosing the global pivotal cohort of BEAM-302, advancing a one-time in vivo base-editing therapy designed to address both lung and liver disease in severe alpha-1 antitrypsin deficiency. Representative image.
Pharma & Biotech

Beam Therapeutics is trying to rewrite a disease-causing mutation inside the liver. Its pivotal test has now begun

Beam Therapeutics has begun dosing in the global pivotal cohort of its BEAM-302 trial, advancing a one-time in vivo base-editing therapy that could address both lung and liver disease in people with severe alpha-1 antitrypsin deficiency.

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A representative image illustrating BEAM-302 base-editing research as Beam Therapeutics begins pivotal testing of its one-time investigational treatment for alpha-1 antitrypsin deficiency affecting the lungs and liver.
Pharma & Biotech

First patient dosed in BEAM-302 pivotal cohort for alpha-1 antitrypsin deficiency

BEAM-302 enters pivotal AATD testing after early base-editing data. Read how the study could support an accelerated approval pathway.

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