International Top Pharmaceutical Awards

The strategy involves delivering genetic information for two types of molecules: a light-sensitive protein known as CoChR that can produce positively charged currents, and a bioluminescence-emitting enzyme. Packed into an altered virus particle and delivered to cancer cells, the proteins are produced as their genes are expressed in mitochondria. A follow-up injection of a specific chemical turns on the enzyme’s light to activate CoChR, which leads to mitochondrial collapse. The other half of the battle is ensuring this therapy does not interfere with normal cells. Liu’s lab specializes in targeted anti-cancer therapy development. The foundation for the delivery system in this work is the well-characterized adeno-associated virus (AAV), a minimally infectious virus engineered to carry genes and promote their expression for therapeutic purposes. The team refined the system to enhance its cancer specificity by adding a promoter protein to drive up expression of the CoChR and biolumine...

New safety information for healthcare professionals Veoza (fezolinetant): new recommendations to minimise risk of liver injury The PRAC has agreed to a direct healthcare professional communication (DHPC) informing of the risk of drug-induced liver injury (DILI) with Veoza (fezolinetant) and has recommended monitoring of liver function before and during treatment. Veoza is a medicine used to treat moderate-to-severe vasomotor symptoms (also referred to as hot flushes or night sweats) associated with menopause. The committee considered a review of the potential risk of fezolinetant to cause drug-induced liver injury based on information from all available sources, including reports of suspected side effectsand studies published in the scientific literature. Severe elevations of the liver enzymes alanine aminotransferase (ALT) and/or aspartate aminotransferase (AST) (>10x upper limit of normal) with concurrent elevations in bilirubin and/or alkaline phosphatase (ALP) have been reporte...

This week: Partnerships to advance cell and gene therapies have been announced, focusing on three key areas: developing gene therapies, enhancing cell and gene therapy manufacturing and developing treatments for difficult-to-treat conditions. The news highlights:Centers of excellence for gene therapy research Advancing cell and gene therapy manufacturing Developing therapies for difficult-to-treat conditions Centers of excellence for gene therapy research The Medical Research Centre (MRC), part of UK Research and Innovation (UKRI; both Swindon, UK) is establishing two Centers of Research Excellence (CoRE) to advance therapies for untreatable diseases, each receiving up to roughly US$64 million over 14 years aiming to transform treatments for diseases such as heart disease, immune disorders and neurodegenerative diseases like Huntington’s disease. Oxford University (UK) will lead the MRC/BHF CoRE in Advanced Cardiac Therapies, which will be co-funded with the British Heart Foundati...

Offering hope to around 136,000 Haemophilia A patients in India, the Department of Biotechnology (DBT), along with the Centre for Stem Cell Research (CSCR) at Christian Medical College (CMC), Vellore, announced that they have successfully executed a first-in-human gene therapy using lentiviral vectors for the disease. Haemophilia is a severe bleeding disorder caused by the deficiency of clotting Factor VIII, leading to spontaneous internal and external bleeding episodes. Although rare, India bears the world’s second-largest burden of haemophilia. Current treatments require frequent Factor VIII replacement therapy, which faces challenges such as high costs, intravenous (IV) access in children, and low patient acceptance.The single-centre study, which enrolled five participants aged between 22 and 41 years, showed transformational results, according to scientists involved in the research. “The therapy successfully produced zero annualised bleeding rates in all five enrolled participan...

AMD has appointed Tim Keating as senior vice president of Government Relations and Regulatory Affairs, effective immediately. His experience is expected to enhance the company’s engagement with key stakeholders as the importance of AI and high-performance chips continues to grow. Ava Hahn, AMD’s senior vice president and general counsel, emphasized Keating’s extensive public policy expertise and understanding of regulatory landscapes will be crucial for the company’s future. Keating brings decades of experience, including a significant tenure at Boeing and roles within the U.S. government. Prior to joining AMD, Keating was the executive vice president at Boeing, and he has also held prominent positions at Honeywell and within the U.S. House of Representatives. He holds a bachelor’s degree in political science from the University of Scranton and an honorary doctorate from the University of South Carolina.

A new kind of cancer gene therapy can be remotely activated at a specific part of the body. The team developed a version of CRISPR that responds to ultrasound, and demonstrated how it can be used to clear cancer in mice.CRISPR is a powerful genetic editing tool that uses an enzyme called Cas9 to make precise edits to targeted genes. The problem is, it doesn’t always stay in the right part of the body, and can continue editing genes long after it’s needed, potentially triggering an immune response.Now, scientists at the University of Southern California (USC) have demonstrated a new way to control when and where CRISPR does its work. In tests in mice, they used it to clear out cancer. In practice, CRISPR could be incorporated into virus delivery vehicles and delivered intravenously to a patient. Then, focused ultrasound pulses can be directed at the desired part of the body, which activates the gene editing tool there and there alone. The trick is that the cells are designed to produ...

Though he doesn't remember it, Branden Baptiste had his first sickle cell crisis at age 2. Through elementary school, he was in and out of the hospital with pain episodes, not knowing why. As he got older, he learned he had sickle cell disease. His red blood cells were forming sickle shapes and getting stuck in his blood stream, preventing oxygen from reaching his tissues. "Sickle cell disease has a broad spectrum of severity, and the severity and frequency of complications can wax and wane," says Matthew Heeney, MD, Branden's long-time hematologist at Boston Children's Hospital. "Unfortunately, Branden was quickly acquiring many of the chronic complications of sickle cell disease, including organ dysfunction affecting his kidneys, lungs, joints, and eyes." Choosing base editing gene therapy Around that time, potentially curative gene therapies for sickle cell disease were becoming available. Heeney, then director of Boston Children's Sickle Cell D...

Investment for companies developing cell and gene therapies (CGTs) was discussed during the GenScript Biotech Global Forum London 2024 conference held on 20 November 2024. Following a sharp drop in CGT investment in 2022, trends suggest that CGT investment is currently undergoing a “renormalisation”. However, investors continue to be selective, said speakers Josh Resnik, senior managing director at RA Capital Management, and Ping Shek, managing director at Stifel’s Healthcare Investment Banking Division. CGTs have faced challenges attracting investment due to the large amount of capital required to manufacture CGTs, as well as hurdles relating to reimbursement of one-off, high-cost therapies and patient access. Resnik noted that investor sentiment about CGTs was impacted by competitive pressures from “not-so-complicated biologics” such as antibody-drug conjugates, radiopharmaceuticals, and T-cell engagers in oncology and immunology. Resnik noted that CGTs must demonstrate a stron...