Denali Delivers ‘Welcome Positive’ for Rare Disease Space With FDA’s Hunter Syndrome Nod

Avlayah is the first Hunter syndrome therapy approved to address the condition’s neurologic complications, according to Tracy Beth Høeg, acting director of the Center for Drug Evaluation and Research.

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Beam’s Base Editor Advances to Pivotal Development on Back of ‘Impressive’ AATD Data

Beam is now in the strongest position of all companies advancing a genetic therapy for alpha-1 antitrypsin deficiency, according to analysts at William Blair.

Sarepta Pushes Past Gene Therapy Debacle as RNA Therapies Show Early Promise

Biomarker data for Sarepta Therapeutics’ RNA programs, licensed from Arrowhead Pharmaceuticals, are “competitive” and “strong,” according to analysts at Jefferies, which projected over $1 billion in peak sales.

Wave Stock Cut in Half on Underwhelming Higher Dose Obesity Data

While participants on a lower dose of Wave Life Sciences’ RNA therapy lost 5.3% total fat at the six-month mark, those receiving the higher dose saw a less than 1% drop at three months.

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Denali Delivers ‘Welcome Positive’ for Rare Disease Space With FDA’s Hunter Syndrome Nod

Beam’s Base Editor Advances to Pivotal Development on Back of ‘Impressive’ AATD Data

Sarepta Pushes Past Gene Therapy Debacle as RNA Therapies Show Early Promise

Wave Stock Cut in Half on Underwhelming Higher Dose Obesity Data

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