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Baseball > Alnylam > Flashcards

Alnylam Flashcards

1
Q

What is a STAR?

A

Strategic Therapeutic Area

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2
Q

What are the three late stage drugs?

A

Vutrisiran, Patisiran, Fitusiran, Lumasiran

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3
Q

How does RNAi work?

A

Small interfering RNAs (siRINAs) as part of an RNA induced silencing complex (RISC), binding to disease causing mrNA and guide their destruction.

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4
Q

What does OXLUMO treat?

A

Primary hyper-oxaluria type 1, to reduce oxalate production

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5
Q

Core values?

A

Commitment to people, innovation, urgency, open culture, passion for excellence.

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6
Q

What does ONPATRRO treat?

A

polyneuropathy caused by hereditary ATTR amyloidosis (tingling, burning pain, weakness, CPT)

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7
Q

What mRNA does ONPATRO target?

A

mutant and wild type TTR (trans thyretin) mRNA

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8
Q

What is the reduction associated with ONPATRO

A

Reduction of serum TTR protein and TTR protein deposits in tissues

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9
Q

What does TTR protein do??

A

Made in liver, move aorund Vitamin A or thryoxine. Its a tetramer, looks like an X.

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