International Top Pharmaceutical Awards

Biopharmaceutical manufacturing largely involves biomolecules whose stability depends on temperature. Antibodies, insulin, and, more recently, messenger RNA (mRNA)-based vaccines are examples of biotherapeutics that require cold storage. This article explores the difficulties with developing stability-enhancing formulations for temperature-sensitive biomolecules and technology innovations that are enabling their formulation. Factors that have been most challenging in developing formulations that enhance stability in temperature-sensitive biomolecules include buffer, pH, and sugar. These are usually the typical factors that need to be evaluated to thermodynamically stabilize monoclonal antibodies (mAbs), notes Joseph Cao, PhD, senior scientist, Analytical and Formulations Development, Bionova Scientific. He explains that there are different types of buffers, sugars, and a range of pH to choose from when developing a proper formulation for mAbs that will prevent aggregation, degrada...

CHAMPAIGN, Ill. — Thanks to a serendipitous discovery and a lot of painstaking work, scientists can now build biohybrid molecules that combine the homing powers of DNA with the broad functional repertoire of proteins — without having to synthesize them one by one, researchers report in a new study. Using a naturally occurring process, laboratories can harness the existing molecule-building capacities of bacteria to generate vast libraries of potentially therapeutic DNA-protein hybrid molecules. The findings are detailed in the journal Nature Chemical Biology. “Two of the most common building blocks in biology are nucleic acids — used for making RNA and DNA — and amino acids, which make up proteins,” said University of Illinois Urbana-Champaign biochemistry professor Satish Nair, who led the study with postdoctoral researcher Zeng-Fei Pei. “We have these two sets of biological molecules that do very different things, and, for decades, chemists have been trying to integrate them into...

Scientists from Harvard Medical School are using artificial intelligence (AI) to solve the problem of finding treatments for rare and neglected diseases. They have developed a new method, called TxGNN, that could help identify new drug candidates for the more than 7,000 rare and undiagnosed diseases. Details of the method are discussed in a Nature Medicine paper titled, “A foundation model for clinician-centered drug repurposing.” To date, just 5–7% of rare and neglected diseases have an FDA-approved drug. The rest remain either untreated and undertreated. AI-based applications like TxGNN can help address this challenge by propelling the discovery of new therapies from existing medicines, offering hope for both patients and clinicians. According to its developers, TxGNN is the first method developed specifically for identifying drug candidates for rare diseases and conditions with no treatments. It identified drugs from existing medicines for more than 17,000 diseases, many of which ...

I’m very proud – and always have been – of the advances that have been made in medicinal chemistry and drug discovery. Tremendous amounts of work (and tremendous amounts of public and private investment) have led us to all sorts of discoveries that have made a real difference in the lives of sick patients, and I am very glad indeed to have been a part of that effort for the last 35 years (and counting!) But pride shouldn’t make us unwilling to admit those areas where our knowledge thins out and disappears. There are several very large, very important blind spots in drug discovery that I hope can be illuminated. In no particular order, I would list: Where’d it go? Our lack of knowledge about where our compounds go inside living cells. We know a fair amount about drug distribution and clearance in whole animals and in whole human patients (although still not enough). But intracellular distribution is usually a black box. Cells are tremendously complex spaces, full of distinct regions a...

  An aggressive cancer in which the bone marrow starts producing large numbers of abnormal blood cells, acute myeloid leukemia is the most common type of acute leukemia in adults. Unfortunately, the overall five-year survival rate for the disease is 30%. MyeloMATCH is looking to change that. A precision-medicine initiative sponsored by the National Cancer Institute, myeloMATCH (Myeloid Malignancies Molecular Analysis for Therapy Choice) is an “umbrella trial,” a group of clinical substudies for people with acute myeloid leukemia or myelodysplastic syndrome. Myelodysplastic syndrome is another blood cancer characterized by cytopenia — a reduction in the number of one or more types of blood cells — that sometimes progresses to acute myeloid leukemia. With more than 100 participating locations, myeloMATCH is the largest coordinated effort in the United States to address these myeloid cancers and includes treatment trials and protocols developed by the Alliance for Clinical T...

We don’t want to reject a batch; we understand the role we play in these supply chains and the development of promising new therapies for patients. But if there is any potential risk for a patient or any risk of regulatory noncompliance, there’s nothing else we can do,” says Erwin Van Streun, Quality Assurance Manager & Qualified Person at Sharp Services. Patient safety comes first and foremost in clinical trials, just as it does with commercial pharmaceuticals. Responsible for certifying pharmaceutical products before they’re used in studies across Europe, Qualified Persons play a critical role in safeguarding patients from potential quality issues in the investigative medicinal product (IMP) manufacturing process. To receive approval from regulatory bodies in the European Union (EU), every pharmaceutical sponsor that produces IMP outside of the EU or United Kingdom (UK) must be able to state that their non-EU or non-UK manufacturing and testing sites comply with EU-Good Manufact...

  NEW YORK – In a first-in-human trial, TORL BioTherapeutics ' investigational antibody-drug conjugate TORL-1-23 demonstrated encouraging, albeit early efficacy in patients with advanced solid tumors, particularly those with claudin 6-expressing ovarian cancer.  The drug involves a claudin-6 (CLDN6)-targeting antibody linked to a monomethyl auristatin E (MMAE) cytotoxic payload.   Gottfried Konecny, a gynecologic oncologist at the University of California, Los Angeles, presented data from a Phase I trial  at the European Society for Medical Oncology Congress in Barcelona on Sunday. The first-in-human trial features dose-escalation and -expansion portions.   The drug's target, the oncofetal protein CLDN6, is highly expressed in certain cancers but not in normal tissue. Konecny demonstrated this during his presentation, based on analysis of 54 types of normal tissues, which lacked CLDN6 expression. Other drug targets such as TROP2, and folate rec...