Scribe Therapeutics Picks Cholesterol-Lowering As First In Vivo Gene Target

The company claims its gene editing tech is the most potent yet – and is joining others in making established cardiovascular disease gene PCSK9 its first therapeutic target

Scribe Therapeutics is one of a new generation of CRISPR-based gene-editing companies, and believes its in vivo CRISPR X-Editing (XE) technology could be the most efficient yet.

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Aldeyra Expects Mid-2025 Refiling Of Reproxalap After Second CRL

 
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Aldeyra’s dry eye candidate reproxalap received a second FDA complete response letter, but the firm expressed confidence about refiling quickly based on two ongoing studies.

Lilly Licenses Sangamo’s Capsid Technology For CNS Gene Therapy

 

Deal Snapshot: Lilly is the third company to sign a licensing deal for STAC-BBB with Sangamo, which also aims to secure a deal for its Fabry disease program in the second quarter.

Samsung Bioepis Reevaluating Approach To Biosimilars

 

The challenging US biosimilar market remains a persistent problem, leading the biosimilar developer to reconsider how it invests in the future while pushing for changes.

Finance Watch: As Stocks Tumble, Offerings Slow, Financial Alternatives Rise

 
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Public Company Edition: Stock valuations are falling due to political, economic and regulatory uncertainty, resulting in fewer large public offerings, more alternative financings and cost cuts. Carisma, Tenaya, BioAtla, Arbutus, Nkarta, Alector and Adaptimmune announced layoffs.

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Roche’s Brain Shuttle Delivers In Alzheimer’s, Moves Ahead To Phase III

 

By allowing it to enter the brain more easily, trontinemab’s brain shuttle brings more patients to ‘amyloid zero’ levels faster, and with fewer brain swelling side effects.

Compass’ Bispecific BTC Contender Meets Phase II/III Endpoint

 
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Compass' bispecific antibody tovecimig hits primary efficacy endpoint in Phase II/III top-line data in advanced biliary tract cancer, and may have class side-effect advantages. But additional survival data may be needed to support US approval.

AZ’s Oncology R&D Head On China’s Scientific Promise And True Innovation

 

AstraZeneca remains committed to investing in R&D and alliances in China, where Susan Galbraith, the UK major’s head of oncology R&D, sees innovation eventually reaching parity with the US and Europe.