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Today's Rundown

Featured Story

Centessa eyes registration in hemophilia as SerpinPC shows reduction of bleeding in phase 2 study

Centessa Pharmaceuticals' hemophilia drug reduced bleeding rates and spontaneous joint bleeds in a midstage study, setting the biotech up to move the candidate closer to regulatory submission. 

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Top Stories

Orchard picks an apple from Novartis' field with new chief technical officer

Gene therapy biotech Orchard Therapeutics has picked a shiny apple from Novartis, bringing Nicoletta Loggia, Ph.D. on as chief technical officer after a 17-year career at the Swiss pharma. Loggia previously served as global head of cell and gene therapies for Novartis—a high profile part of the pharma's business that in recent years has launched Zolgensma.

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Cardiff reveals early data on challenger to Amgen's KRAS crown

Amgen has already won the KRAS race by getting Lumakras across the finish line, but Cardiff Oncology has just rolled out some early data for a follow-on candidate showing partial responses and extended survival without disease progression compared to standard of care.

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Vor’s cancer cell therapy gets on FDA fast track ahead of 2022 data drop

The FDA has become the latest organization to validate the potential of Vor Biopharma’s approach to CRISPR-powered cell engineering, granting fast-track status to its lead asset shortly after Johnson & Johnson struck a deal to access the technology.

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Ixaka’s cell therapy clears early phase 3 data review

Cells derived from bone marrow have shown some promise in the treatment of patients with badly blocked arteries over the past 20 years without ever landing a knockout blow on the condition. Now, Ixaka has edged closer to showing if its prospect REX-001 can buck the trend by coming through an interim data analysis.

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As gene therapy safety faces scrutiny, Broad Institute proposes a safer and more efficient delivery tool

A team led by scientists at the Broad Institute has developed adeno-associated viruses as gene therapy vectors. The vehicles can target muscle tissue, and therapies based them are effective in animals at low doses, potentially reducing the risk of liver damage, they said.

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Boehringer Ingelheim, Twist Bioscience team up for $710M+ antibody discovery collab

Boehringer Ingelheim will look to Twist’s “Library of Libraries” of antibody sequences to single out potential treatments for drug targets.

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Despite mixed phase 3 showing, Apellis hopes to push eye disorder drug past FDA's finish line

Apellis Pharmaceuticals’ pegcetacoplan posted mixed results in two late-stage trials studying the C3 inhibitor in patients living with geographic atrophy (GA). Despite the mixed showing, CEO Cedric Francois thinks the med's performance was strong enough to warrant an FDA nod.

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Biogen CEO blames Aduhelm’s rocky launch on 'too much confusion, misinformation and controversy'

Before Biogen scored an FDA approval for Alzheimer's drug aducanumab, the company probably hoped its launch would go much smoother than the last three months have been. It turns out Biogen's CEO Michel Vounatsos is willing to say so publicly.

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Fierce Pharma Asia—Takeda's AbbVie drug supply lawsuit, Novavax' COVID vaccine deal; Juno, WuXi's CAR-T nod

A court says AbbVie can't force Takeda to fulfill Lupron supply obligations. Takeda will make 150 million doses of Novavax's COVID-19 vaccine for Japan. A joint venture between Juno Therapeutics and WuXi AppTec won China approval for a Breyanzi sister CAR-T therapy. And more.

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Chutes & Ladders—Former FDA Commissioner Hahn takes interim CMO role at YourBio Health

Former FDA Commissioner Stephen Hahn is joining YourBio Health as interim chief medical officer. Gilead's global head of liver diseases, Tram Tran, M.D., will be Glympse Bio's first chief medical officer. And Orchard Therapeutics picks an apple from Novartis' field, snagging cell and gene therapies leader Nicoletta Loggia, Ph.D., as chief technical officer.

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Resources

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Choose the right AAV platform for your viral vector-based therapy

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eBrief: Drug Product Process Development: Ensuring a Consistent, High-quality Biologic

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Whitepaper: Achieving a Successful Drug Product Technology Transfer

Get insights on key considerations for a successful tech transfer process for manufacturing a biologic drug product, including a case study on overcoming challenges in a process transfer for a sterile diluent.

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