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5 days ago | 
themedicinemaker.com | Stephanie Vine
Researchers are exploring a combined approach using gene therapy and nanotechnology to address diabetic retinopathy (DR), a leading cause of vision loss among adults. A new review, led by scientists at Curtin University and collaborators from Australia, New Zealand, and Ethiopia, evaluated recent progress in bio-nanotechnological tools for delivering gene therapies to the retina, with the goal of improving treatment specificity, longevity, and safety.
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2 weeks ago | 
themedicinemaker.com | Stephanie Vine
“For me personally, this project is very close to my heart. I spent about a decade at Novartis and GSK, and I’ve brought that experience to Gates MRI to focus on diseases that disproportionately affect underserved populations and present significant challenges to global health. It’s an exciting time.
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2 weeks ago | 
themedicinemaker.com | Stephanie Vine
Stanford University researchers have developed a machine learning-based approach to engineer safer and more effective proteins for use in cell and gene therapies. The work centers on using zinc fingers – tiny, naturally occurring human proteins involved in gene regulation. Xiaojing Gao, senior author of the paper and assistant professor of chemical engineering at Stanford explains: “In this paper, we raise the question: Why not design treatments that avoid immune reactions from the start?
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2 weeks ago | 
themedicinemaker.com | Stephanie Vine
US Secretary of Health and Human Services (HHS) RFK Jr has removed all 17 members of the CDC’s Advisory Committee on Immunization Practices (ACIP), claiming that it was “plagued with conflicts of interest.” The committee’s activities would typically include reviewing vaccine data and making recommendations about who should be eligible for vaccines, as well as what insurers should cover. A statement from HHS said: “A clean sweep is necessary to reestablish public confidence in vaccine science.
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3 weeks ago | 
themedicinemaker.com | Stephanie Vine
According to West’s Laura Englander, “Drug substance manufacturing, which precedes the fill-finish step, is a complicated process, especially when it comes to biologics. In comparison, fill-finish can be seen as a simpler process with less complexity, but there are many technical aspects to consider to ensure quality, sterility and safety.”Mistakes in fill-finish can lead to project delays, increased costs – and even company failure.
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3 weeks ago | 
themedicinemaker.com | Stephanie Vine
A study by researchers at the San Raffaele Telethon Institute for Gene Therapy (SR-Tiget) has outlined a potentially scalable strategy for in vivo gene therapy targeting hematopoietic stem and progenitor cells (HSPCs). The work identifies a developmental window shortly after birth during which circulating HSPCs can be accessed directly in the bloodstream and genetically modified without the need for myeloablative conditioning or transplantation.
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3 weeks ago | 
themedicinemaker.com | Stephanie Vine
Researchers at Washington University School of Medicine have investigated how different preconditioning strategies affect the performance of dendritic cell (DC) vaccines in a mouse model of cancer. Their findings show how low-dose radiation may enhance therapeutic outcomes. Preconditioning regimens, such as lymphodepleting chemotherapy or radiation, are routinely employed to improve the efficacy of adoptive cell therapies.
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3 weeks ago | 
themedicinemaker.com | Stephanie Vine
The FDA has placed on hold a phase II trial of Rocket Pharmaceuticals’ RP-A501, an investigational gene therapy for Danon disease, following a patient fatality. The individual experienced a serious adverse event involving capillary leak syndrome, later complicated by an acute systemic infection that resulted in death. Rocket is conducting a full root cause analysis, with a particular focus on a recently introduced immunosuppressive agent added to the pre-treatment protocol.
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3 weeks ago | 
themedicinemaker.com | Stephanie Vine
How much attention do you pay to the fill-finish process? And how much do you know about its importance? The fill-finish step is the final and one of the most critical stages of the production process, where the drug product – often a sterile liquid – is transferred into its final container, such as a vial, syringe, or cartridge. For lyophilized drugs, the liquid is filled into the container and then freeze-dried in place.
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1 month ago | 
themedicinemaker.com | Stephanie Vine
Antibody drug conjugates – hype or hope? According to Catherine Bladen, Vice President, Regional Executive and Principal Advisor of Vector Laboratories, there’s a wealth of exciting advances shaping the development space of antibody drug conjugates (ADCs). We get her view in this quick-fire Q&A. For me, it’s the opportunity to be involved in research that changes patients' lives in real time. ADCs have revolutionized therapeutic strategies by targeting multiple antigens or epitopes.
