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STAT+: Astellas retries XLMTM gene therapy after deaths

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New hair loss data for a sort of amped-up minoxidil is looking very plush. Also Astellas is reentering the clinic with a next-generation gene therapy for XLMTM and Intellia’s CRISPR therapy for hereditary angioedema impresses.

The need-to-know this morning

  • Eli Lilly said it is buying Ajax Therapeutics, a privately held developer of blood cancer drugs, for as much as $2.3 billion. Ajax’s lead medicine is a next-generation JAK2 inhibitor currently being evaluated in a Phase 1 study in patients with myelofibrosis.
  • Ligand Pharmaceuticals said it is buying Xoma, an aggregator of drug royalties, for $739 million.  
  • Compass Therapeutics said its drug for advanced biliary cancer delayed tumor progression but failed to prolong survival compared to chemotherapy in a clinical trial. 
  • BridgeBio shares were higher this morning on reports from the weekend that Pfizer had settled two of three patent cases regarding Vyndamax, its medicine for the heart condition known as ATTR-CM. While terms of the settlement have not been disclosed, any extension of patent protection for Vyndamax is also positive for BridgeBio, which sells a competing ATTR-CM drug called Attruby.

CRISPR therapy slashes rare swelling attacks

Intellia Therapeutics said this morning that its one-time CRISPR treatment lonvo-z cut attack rates by 87% in a Phase 3 trial of hereditary angioedema, STAT’s Jason Mast writes. With more than 60% of patients becoming attack-free and no serious safety signals reported, the therapy is being positioned as a functional cure — or at least a major upgrade over existing options.

Continue to STAT+ to read the full story…

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Want to stay on top of the science and politics driving biotech today? Sign up to get our biotech newsletter in your inbox.

New hair loss data for a sort of amped-up minoxidil is looking very plush. Also Astellas is reentering the clinic with a next-generation gene therapy for XLMTM and Intellia’s CRISPR therapy for hereditary angioedema impresses.

The need-to-know this morning

  • Eli Lilly said it is buying Ajax Therapeutics, a privately held developer of blood cancer drugs, for as much as $2.3 billion. Ajax’s lead medicine is a next-generation JAK2 inhibitor currently being evaluated in a Phase 1 study in patients with myelofibrosis.
  • Ligand Pharmaceuticals said it is buying Xoma, an aggregator of drug royalties, for $739 million.  
  • Compass Therapeutics said its drug for advanced biliary cancer delayed tumor progression but failed to prolong survival compared to chemotherapy in a clinical trial. 
  • BridgeBio shares were higher this morning on reports from the weekend that Pfizer had settled two of three patent cases regarding Vyndamax, its medicine for the heart condition known as ATTR-CM. While terms of the settlement have not been disclosed, any extension of patent protection for Vyndamax is also positive for BridgeBio, which sells a competing ATTR-CM drug called Attruby.

CRISPR therapy slashes rare swelling attacks

Intellia Therapeutics said this morning that its one-time CRISPR treatment lonvo-z cut attack rates by 87% in a Phase 3 trial of hereditary angioedema, STAT’s Jason Mast writes. With more than 60% of patients becoming attack-free and no serious safety signals reported, the therapy is being positioned as a functional cure — or at least a major upgrade over existing options.

Continue to STAT+ to read the full story…

Read More

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From Sequence to Patient in Under 12 Months: A Case Study in Advancing Complex Cancer Immunotherapies

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Image of Joseph Shultz

Joseph Shultz

Vice President of Technical Development and Manufacturing
Ottimo Pharma

Panelist

Image of Joseph Shultz

Joseph Shultz

Joseph Shultz is the vice president of technical development and manufacturing at Ottimo Pharma. His more than 30 years in the industry span development, manufacturing, quality, and technology development. He has held influential positions at Amgen, Novartis Pharma, the Battelle Memorial Institute, Evelo Biosciences, and Resilience. He initiated the technologies and led the strategies that resulted in next-generation biomanufacturing plants at both Amgen and Novartis.


Image of Imroz Ghangas

Imroz Ghangas

Vice President of Commercial Sales
Asimov

Panelist

Image of Imroz Ghangas

Imroz Ghangas

Imroz Ghangas and his team drive partnerships to advance Asimov’s genetic design platform and AI capabilities. With over 25 years in biotech, Imroz has evolved from process development scientist to commercial leader, bridging technical innovation with scalable solutions. His expertise spans bioprocess development and platform integration, with deep knowledge of biomanufacturing workflows from gene to drug product. He leverages his technical foundation to accelerate the adoption of next-generation bioprocessing technologies.


Broadcast Date: 

  • Time: 

Complex biologics such as bifunctional antibodies are opening new therapeutic possibilities in oncology, but these molecules present significant challenges for manufacturing teams. Non-standard architectures can often translate to low expression and difficult developability, making cell line development a critical bottleneck between a promising sequence and a viable clinical candidate.

In this GEN webinar, Joseph Shultz (vice president of technical development and manufacturing, Ottimo Pharma) and Imroz Ghangas (vice president of commercial sales, Asimov) discuss strategies for achieving high-performing clonal titers and advancing a dual-paratopic cancer immunotherapy from sequence to dosed patient in under a year. Attendees will learn about the unique attributes of Ottimo’s molecule and how a specialist partnership with Asimov accelerated the program. The presenters will also introduce the CHO Edge System, which combines Asimov’s proprietary GS knock-out CHO host, hyperactive transposase, library of characterized genetic elements, and AI-driven genetic design tools to routinely deliver clonal titers of 8-12 g/L.

A live Q&A session will follow the presentation offering you a chance to pose questions to our expert panelists.

Produced with support from:

asimov logo

The post From Sequence to Patient in Under 12 Months: A Case Study in Advancing Complex Cancer Immunotherapies appeared first on GEN – Genetic Engineering and Biotechnology News.

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G-Link CAR-T Delivery Platform Showcased at ASGCT

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Vyriad reports that it will showcase its latest CAR T delivery technology platform, G-Link, through a schedule of presentations, technical sessions, and exhibition activities at ASGCT. The modular plug-and-play protein adapter developed in collaboration with Menachem Rubenstein, PhD, of the Weizmann Institute allows drug developers to cap and retarget existing lentiviral vectors for in vivo delivery, according to the company.

By leveraging G-Link, wild-type lentiviral vectors can be reprogrammed for in vivo applications without the need for intensive vector re-engineering, effectively shortening development timelines for next-generation CAR T and other cell therapies, notes a company spokesperson, who adds that G-Link can also be used to simplify ex vivo CAR T manufacturing and significantly improve T cell transduction efficiency without redesigning vectors.

“I believe that G-Link can address some of the most persistent challenges in in vivo delivery and we are excited to unveil it at ASGCT this year,” says Stephen Russell, PhD, CEO of Vyriad. “Our participation this year underscores our clear mission: to replace complex, weeks-long manufacturing cycles with precise, off-the-shelf immunotherapies. With G-Link, we aim to foster collaborations that will define the next generation of in vivo cell therapies.”

Vyriad’s VV169 in vivo CAR T program will progress into clinical development later this year, while the G-Link platform will advance towards clinical translation later in the future, continues Russell.

 

The post G-Link CAR-T Delivery Platform Showcased at ASGCT appeared first on GEN – Genetic Engineering and Biotechnology News.

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Sarepta tumbles as its gene therapy sales decline further

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Even though Elevidys beat Wall Street expectations, analysts expect investor focus to shift toward Sarepta’s early-stage RNA drugs.

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