Pharma Veterans

Two Landmark New Drug Approvals – Asrar Qureshi’s Blog Post #897 Dear Colleagues!  This is Asrar Qureshi’s Blog Post #897 for Pharma Veterans. Pharma Veterans  aims to share knowledge and wisdom from Veterans for the benefit of Community at large. Pharma Veterans Blog is published by Asrar Qureshi on  WordPress, the top blog site. Please email to asrar@asrarqureshi.com for publishing your contributions here. Credit: Google Images Credit: Google Images Credit: Google Images In December 2023, US Food and Drug Administration approved two gene therapy-based drugs for sickle cell disease treatment.  CASGEVY (exagamglogene autotemcel) is the first approved CRISPR/Cas9 genome-edited cell therapy for the treatment of Sickle Cell Disease – SCD, in patients 12 years or older with frequent vaso-occlusive crises (VOCs). CASGEVY has been developed by Vertex Pharmaceuticals and CRISPR Therapeutics and has been shown to reduce or eliminate vaso-occlusive crises for SCD patients....

New Gene Therapy Products – Part 2 – Asrar Qureshi’s Blog Post #747 Dear Colleagues!  This is Asrar Qureshi’s Blog Post #747 for Pharma Veterans. Pharma Veterans welcome sharing of knowledge and wisdom by Veterans for the benefit of Community at large. Pharma Veterans Blog is published by Asrar Qureshi on  WordPress, the top blog site. Please email to asrar@asrarqureshi.com for publishing your contributions here. Photo Credit: Gilmer Diaz Estela Photo Credit: Thirdman Photo Credit: Tiger Lily Gene therapy is among the newest modalities of treatment. It is still an experimental treatment that works by introducing genetic material into a person’s cell to fight or prevent disease. Gene Therapy is presently being tested for several diseases, such as autoimmune disorders, hemophilia, thalassemia, Parkinson’s disease, cancer, diabetes, heart disease, and so on. Gene therapy drugs are to be given as a single dose, only once in life.  We continue to look at some more drugs. CARVY...

Gene Therapy Progress III – Pharma Veterans’ Blog Post #520 by Asrar Qureshi Dear Colleagues!  This is Pharma Veterans’ Blog Post #520. Pharma Veterans welcome sharing of knowledge and wisdom by Veterans for the benefit of Community at large. Pharma Veterans Blog is published by Asrar Qureshi on WordPress, the top blog site. Please email to asrar@asrarqureshi.com for publishing your contributions here. Continued from Previous…… It would have become clear by now that the most important link in the chain of gene therapy is the ‘Vector’. Vectors transport the genes or gene-modified cells to the target site in human body. We shall conclude this topic with a discussion on Vectors and the challenges faced by the gene therapy. In the earlier part, I had mentioned that viruses are the most favored vectors. McKinsey published a report recently about the ‘promise of viral vectors’, and I shall partake some portion from there. I shall try to stay away from heavy technicalities and keep it sim...

Gene Therapy Progress II – Pharma Veterans’ Blog Post #519 by Asrar Qureshi Dear Colleagues!  This is Pharma Veterans’ Blog Post #519. Pharma Veterans welcome sharing of knowledge and wisdom by Veterans for the benefit of Community at large. Pharma Veterans Blog is published by Asrar Qureshi on WordPress, the top blog site. Please email to asrar@asrarqureshi.com for publishing your contributions here. Continued from Previous…… The Human Genome Project, which ran from 1990 to 2003, provided researchers with the basic information about the genetic content of human organism. The discovery opened many new avenues and opportunities in various fields. Healthcare could also be a great beneficiary of genetic research. National Human Genome Research Institute, working as part of NIH in USA is focused on application of genome knowledge in healthcare. Researchers at the NHGRI are working with patients and families to better understand how genes can cause or influence diseases and develop new ...

Gene Therapy Progress I – Pharma Veterans’ Blog Post #518 by Asrar Qureshi Dear Colleagues!  This is Pharma Veterans’ Blog Post #518. Pharma Veterans welcome sharing of knowledge and wisdom by Veterans for the benefit of Community at large. Pharma Veterans Blog is published by Asrar Qureshi on WordPress, the top blog site. Please email to asrar@asrarqureshi.com for publishing your contributions here. Gene therapy may be much less known in Pakistan, but it is progressing rapidly in the developed world. Owing to the vast potential and promise it holds, Big Pharma and Small Biotech companies are racing to find new therapies. Big Pharma has deep pockets of its own, while Small Biotech are being liberally funded by VCs - Venture Capital firms. Promises of gene therapy are mainly two: one, the possibility to treat some yet untreatable diseases; two the potential to make unprecedented amounts of money. We shall focus on the innovation for good part only. Gene Therapy “Gene therapy is a te...

Dear Colleagues!  Today is Pharma Veterans Blog Post #153. Pharma Veterans shares the wealth of knowledge and wisdom of Veterans for the benefit of entire Pharma Community. It aims to recognize and celebrate the Pharma Industry Professionals.  Pharma Veterans Blog  is published by Asrar Qureshi on WordPress , the top blog site. If you wish to share your stories, ideas and thoughts, please email to asrar@asrarqureshi.com for publishing your contributions here . The following information is based on Reuters/ BBC reporting. Professor He Jiankul, Associate Professor in the Southern University of Science and Technology in the southern city of Shenzhen announced recently that he had performed gene editing to help protect two babies from future infection with AIDS virus. He released videos on YouTube on Monday and issued a statement that he used gene-editing technology known as CRISPR-Cas9 to edit the genes of twin girls to create the first gene-edited babies. Th...