The Medicine Maker

The creation and production of small molecules, biologics, and cutting-edge therapies rely on the skills, dedication, and expertise of a diverse group of professionals. At The Medicine Maker, we highlight these individuals, sharing success stories from the industry while also addressing its significant challenges. Our engaging content spans the full range of drug development, ensuring that everyone involved in medicine stays informed about the latest issues, trends, and innovations shaping the pharmaceutical sector. Additionally, The Medicine Maker team offers The Small Molecule Manufacturer, a platform dedicated to applauding the people, processes, and technologies that are revolutionizing the development and manufacturing of small molecule drugs.

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Gene Therapy Meets Nanotechnology

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.
At the Front Lines of Tuberculosis Vaccine Research With the Gates Medical Research Institute

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.
Using Machine Learning to Improve Gene Therapy Design

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?
RFK Jr Fires CDC Vaccine Advisory Committee

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.
Navigating the Therapeutic Landscape of GERD

3 weeks ago | themedicinemaker.com | Rob Coker

Did you know that gastroesophageal reflux disease (GERD) remains one of the most prevalent gastrointestinal disorders worldwide, with significant implications for healthcare systems and patient quality of life? Understanding the evolving landscape of GERD pathophysiology, diagnosis, and treatment can help identify therapeutic gaps and innovation opportunities. GERD is a common but complex condition.
This Week’s Biopharma News: Argentina’s WHO Exit and Sanofi Acquisition of Blueprint Medicines

3 weeks ago | themedicinemaker.com | Rob Coker

Sanofi intends to acquire Blueprint Medicines in a transaction valued at up to $9.5 billion, comprising $129.00 per share in cash and a contingent value right of up to $6.00 per share tied to regulatory milestones. The acquisition will give Sanofi full rights to Ayvakit (avapritinib), currently the only approved treatment for both advanced and indolent systemic mastocytosis, which generated $479 million in revenue in 2024.
Preparing National Strategies for CAR T: The Latest IQVIA Report

3 weeks ago | themedicinemaker.com | Rob Coker

The field of oncology has witnessed a tectonic shift in recent years, driven by the rise of advanced therapies. At the forefront are chimeric antigen receptor T-cell (CAR T) therapies – personalized, living drugs that have redefined treatment paradigms for hematological malignancies. As their clinical efficacy garners headlines, however, a more sobering reality persists: health systems are not yet fully equipped to deliver on their promise.
Worst-Case Scenarios and Ideal Facilities in Fill-Finish

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.
Gene Therapies for Newborns

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.
Breakthrough Treatments for Psoriasis Offer New Hope for Patients

3 weeks ago | themedicinemaker.com | Rob Coker

Veteran musical icon Art Garfunkel recently shared the story of his lifelong struggle with psoriasis for the first time. Throughout his career, he managed to conceal the condition using various treatments, including creams and UV light therapies, which provided only temporary relief. The persistent discomfort eventually led him to step back from performing during his legendary partnership with songwriter Paul Simon.