The previous year witnessed many promising developments in the fight against rare diseases. The recent Casgevy's FDA approval for beta thalassemia comes just one month after the US drug regulator approved the use of Casgevy in treating sickle cell disease — which is notably the first time the CRISPR-based treatment received a regulatory green light in the United States.
Casgevy uses the novel CRISPR gene-editing technology to modify patients’ blood cells and transplant the modified cells back into the bone marrow, triggering an increase in the production of haemoglobin, according to the FDA.
Rare diseases are considered as a significant health challenge. Between 5,000 and 8,000 rare diseases have been identified and these diseases, though not prevalent like other diseases, as a group, affect 6 to 8 per cent of the global population. Rare diseases present unique problems for not only the individuals living with the rare medical condition but for caregivers, researchers, policymakers, and industries as well.
More than 80 per cent of rare diseases are caused by genetic or congenital aberrations, and 75 per cent present with a wide range of neurological symptoms and physical and intellectual disabilities. Sadly, rare diseases mostly affect children or young adults, and even worse, several siblings can be affected in the same family. As such, these diseases come with substantial hardship for both parents and patients. Hence, advancing therapies to cater to their wellbeing is of utmost critical.
Urgency for R&D
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Denne historien er fra BioSpectrum Asia Feb 2024-utgaven av BioSpectrum Asia.
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How To Control Indian Diabetes Growth
World Diabetes Day, observed on November 14, reminds us of the growing impact of diabetes on global health and the urgent need for collective action to prevent, diagnose, and manage this chronic condition. In 2024, the theme 'Breaking Barriers, Bridging Gaps' underscores the critical importance of accessible, high-quality diabetes care, especially for underrepresented communities. The Government of India has taken many measures intending to reduce the prevalence of diabetes and alleviate its long-term complications, contributing to a healthier future for all citizens.
HIV Prevention Remains Elusive
On December 1, the world gathers every year to commemorate World AIDS Day, which serves as an important reminder that we must remain steadfast in our commitment to prevent new HIV (Human Immunodeficiency Virus) infections and provide essential services to all people living with HIV globally. In 2024, the 37th World AIDS Day opens with the theme, \"Collective Action: Sustain and Accelerate HIV Progress.\"
Karnataka launches India's first GCC policy
Bengaluru Tech Summit 2024
Innovations in Urological Imaging: Transforming Patient Care in India
Despite advancements in urological imaging, challenges persist in India, particularly regarding cost and accessibility. Government initiatives and public-private partnerships should expand access to imaging facilities. Continued research investment is crucial for developing cost-effective solutions.
How strategic angel investment is driving global expansion in medtech and diagnostics
India has firmly established itself as a key player in the global medtech and diagnostics sectors. With a burgeoning healthcare ecosystem and a growing focus on innovation, the country offers a fertile ground for startups looking to scale their solutions both domestically and internationally. Central to this growth is the role of strategic angel investment, which not only provides financial backing but also offers critical mentorship and strategic direction to help these companies expand their reach.
What's dampening the GLOBAL AMR BATTLE?
Although there have been significant national and worldwide efforts to provide financial incentives for antibiotic research and development, it is still unclear how best to fortify the existing programmes to further spur antibiotic innovation. The incentive programmes in place now are a crucial first step in enhancing the economic viability of antibiotic development. However, it seems like there isn't enough global coordination among all the programmes, which could lead to duplication of effort, funding gaps in the value chain, and the failure to include crucial AMR objectives. Let's dig deeper.
Indian Lifesciences Industry Going Ahead in its Innovation Journey While Navigating Challenges
While India is a hub for biosimilar manufacturing and continues to build its capabilities to manufacture these complex therapeutics, Indian biopharma companies are building R&D capabilities and establishing public-private collaborations and Centre of Excellences (CoEs) to develop innovative biologics and advanced therapeutic modalities like cell and gene therapy (CGT) and mRNA vaccines and therapies, biopharma sector remained robust in 2023, accounting for $53.8 billion (35 per cent of the BioEconomy). The growth was primarily driven by advances in biotechnology, personalised medicine innovations, and an expanding market for biosimilars in 2024.
BioPharma's Transformative & Strategic OUTLOOK 2025
The Indian biopharma industry witnessed notable transformative developments and announcements, marked by innovation, regulatory advancements, and market expansion this year. From achieving global recognition in biosimilars to advancing biomanufacturing capabilities and precision therapeutics, the sector has solidified its role as a key player in the global pharmaceutical landscape. This progress underscores India's strategic focus on leveraging its robust infrastructure, skilled talent pool, and government support to meet the evolving demands of the healthcare industry.
Promega unveils GloMax Galaxy Bioluminescence Imager for illuminating protein dynamics in real time
US-based Promega Corporation, a life-sciences research partner dedicated to providing intuitive tools that empower scientists to innovate, has unveiled the new GloMax Galaxy Bioluminescence Imager.
Singleron and Bioscreen to enhance access to single cell multi-omics solutions for Indian researchers
Singleron Biotechnologies, a leading innovator in single cell multi-omics solutions, has announced a strategic partnership with Bioscreen, a renowned distributor of advanced life science products in India.
