International Top Pharmaceutical Awards

What physico-chemical properties do your formulas have? Will they remain stable? Do their viscosities, performance, etc., endure changes in pH or temperature?Physico-chemical property analysis is one way to predict their behaviors. As ingredient supplier Seppic explains on its website, "physico-chemical tests are used to characterize properties, anticipate behavior in formulation and measure performance . .. under real usage conditions." The company adds that such tests also help to optimize the ingredient or formula structure, guiding the product developer to make adjustments that achieve the functionality and behavior desired by end users.More specifically, as Calixto and Maia Campos report, rheological, texture and sensory analyses are important to understanding the behavior and stability of formulations. Cheung, et al., even proposed the creation of products based on their constituents and microstructure, rather than traditional trial-and-error and screening formula var...

Model organisms have played an indispensable role in advancing our understanding of neurological disorders and developing therapeutic interventions. Traditionally, mammalian models , particularly rodents, have dominated this research space. However, non-mammalian models such as Caenorhabditis elegans (C. elegans), Drosophila melanogaster (D. melanogaster), Lymnaea stagnalis (pond snails), and Danio rerio (zebrafish) are gaining recognition due to simpler anatomy, shorter life cycles, and genetic manipulability. Moreover, their ability to exhibit various forms of learning and memory makes them suitable model organisms for neuropharmacological studies aimed at investigating the effects of different drugs, bioactive compounds, and neurotoxins on cognitive functions.These models provide valuable insights into the molecular and cellular mechanisms underlying neurological functions and disorders, offering complementary perspectives to those obtained from mammalian studies. Despite thes...

The field of gene therapy has seen unprecedented growth in recent years, with 20 approved indications in the U.S., a record number of approvals in 2023, and a rapid expansion of investigational trials across various diseases. Great strides have been made in potential treatments for inherited and acquired genetic diseases, including hemophilia B, sickle cell disease, and Duchenne muscular dystrophy. But these advances have been accompanied by challenges that have proved unexpectedly difficult to overcome. In the area of rare diseases, in particular, companies have pulled back on their gene therapy ambitions due to safety and efficacy concerns coupled with the high cost of developing and manufacturing these therapies. Viral vector challenges Adeno-associated viruses (AAVs) are currently the most common vectors used for gene delivery. The benefits of AAVs as vectors are well-documented. Wild type AAV is not known to be associated with any human diseases and AAV vectors exhibit broad trop...

Using high-resolution cryo-electron microscopy (cryo-em), researchers from Durham University, Jagiellonian University, and the John Innes Centre uncovered unprecedented detail of gyrase’s action on DNA. The research could aid in the development of next-generation antibiotics that are more precise and effective in stopping bacterial infections. Their findings are published in Proceedings of the National Academy of Sciences (PNAS) in an article titled “Structural basis of chiral wrap and T-segment capture by Escherichia coli DNA gyrase.” DNA gyrase is a bacterial enzyme that introduces negative supercoils into DNA, which helps regulate DNA topology. It is present in bacteria but absent in humans and plays a crucial role in supercoiling DNA, a necessary process for bacterial survival. The enzyme wraps DNA in a “figure-of-eight” loop, then precisely breaks and passes strands through each other, resealing them afterward. This is a delicate process—if the DNA remained broken, it would b...

Efforts to develop a gene therapy for Diamond-Blackfan anemia (DBA) — a rare, life-threatening disorder in which bone marrow cannot make mature, functioning red blood cells — have been hampered by the fact that at least 30 different genetic mutations can cause the disorder. A team led by researchers at Harvard Medical School has now cleared that obstacle by developing a universal gene therapy for DBA, one designed to correct the bone marrow defect no matter the patient’s specific mutation. Get more HMS news here The experimental therapy is ready to test in clinical trials, the team reports Nov. 11 in Cell Stem Cell. “This is one of first examples where we can develop a gene therapy that can target dozens of mutations with a single vector,” said senior author Vijay Sankaran, the HMS Jan Ellen Paradise, MD Professor of Pediatrics at Boston Children’s. All roads lead to GATA1 Children with DBA, which was first described in 1938 at Boston Children’s, have few treatment options. A handfu...

ike many novel therapies, gene therapies are not without challenges that can be solved through a combination of policy and market changes. In order to improve patient access to these life-saving therapies new payment solutions, education efforts around the availability of these treatments and their benefits and risks, and the use of appropriate value assessment methods are all needed, according to a white paper from the National Pharmaceutical Council (NPC).There are a total of 14 gene therapies are now available for hemophilia, sickle cell disease and some cancers, and these potentially curative therapies often only require a single treatment, which can be complicated to deliver. The finances of paying for costly gene therapies is a challenge in the U.S., as well. Lenmeldy (atidarsagene autotemcel) is the most expensive gene therapy, with a price tag of $4.25 million. The therapy is approved to treat metachromatic leukodystrophy, a rare disorder that damages the nervous system. The l...

A patient death in a clinical trial usually prompts the FDA to impose a clinical hold. But Neurogene is able to avoid a lengthy clinical trial pause largely because of its participation in an FDA pilot program intended to speed up the development of therapies for rare diseases. The clinical trial participant who was hospitalized for severe complications after receiving the high dose of Neurogene’s experimental Rett syndrome gene therapy has died. Neurogene disclosed the death in a regulatory filing after the market close Thursday. The company said the FDA is permitting the Phase 1/2 study to continue with the low dose of the therapy, code-named NGN-401. Neurogene added that it will incorporate this dose in planning the design of the clinical trial expected to support a regulatory submission seeking FDA approval of the gene therapy. Rett syndrome is a neurodevelopmental disorder stemming from a mutation in one copy of the MECP2 gene. Neurogene’s NGN-401 delivers to cells a functioning...

Study Rundown: In X-linked adrenoleukodystrophy , pathogenic variations in ABCD1 impair the function of the peroxisomal transporter ATP-binding cassette domain 1 (ABCD1), leading to the accumulation of saturated very-long-chain fatty acids in the body. Approximately 30% of individuals with this condition develop cerebral adrenoleukodystrophy, characterized by inflammation and demyelination of white matter. As a result, the disease is associated with significant cognitive and neurological decline, often leading to early death. Previous research has suggested that elivaldogene autotemcel (eli-cel) gene therapy comprising autologous hematopoietic stem cells transduced with Lenti-D lentiviral vectors with ABCD1 complementary DNA may help prevent disease progression. This study aims to evaluate the efficacy and safety of this treatment and describe its long-term effects, employing an initial 24-month study followed by a subsequent 13-year follow-up period. Most patients survived without ...