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Synlogic pulls plug after peek at phenylketonuria phase III data

Synlogic Inc. jolted Wall Street with news that the firm is scrapping for futility Synpheny-3, its pivotal study with labafenogene marselecobac (SYNB-1934) for phenylketonuria, and will evaluate strategic options. Shares of the Cambridge, Mass.-based firm (NASDAQ:SYBX) fell 48.7%, or $1.68, to end Feb. 9 at $1.77. Synlogic will cease operations and reduce its workforce by more than 90%, retaining only certain employees to help with the wind-down. ....

United Kingdom , Synlogic Inc , Wall Street , Synlogic Inc , Labafenogene Marselecobac , Synb 1934 , Endocrine Metabolic ,

Next-generation genome editing tools surpass CRISPR milestone

Modifying a patient’s DNA is no longer just for science fiction novels. The CRISPR gene editing technique developed by Jennifer Doudna and Emmanuelle Charpentier only took 10 years to reach the market as Casgevy (exagamglogene autotemcel/exa-cel, Vertex Pharmaceuticals Inc.), treating congenital pathologies such as β-thalassemia and severe sickle cell disease (SCD). But science does not stop. ....

Jennifer Doudna , Emmanuelle Charpentier , Vertex Pharmaceuticals Inc , Sickle Cell Disease , Spinal Muscular Atrophy , Base Editing , Prime Editing ,