Pharma Veterans

New Drug Modalities – Asrar Qureshi’s Blog Post 1047 Dear Colleagues! This is Asrar Qureshi’s Blog Post 1047 for Pharma Veterans. Pharma Veterans Blogs are published by Asrar Qureshi on its dedicated site https://pharmaveterans.com. Please email to pharmaveterans2017@gmail.com for publishing our contributions here. Credit: Edward Jenner Credit: Ivan Samkov Credit: Jonathan Borba This post is based on Boston Consulting Group article authored by Lu Chen, Brian Bush, Mike Brochu, and Gian King. Link at the end. Preamble The second annual report from BCG reviews the growth of new drug modalities. The report tracks the progress in each of the six categories of novel modalities in terms of number of products and pipeline revenue. It will help to see which modalities are progressing more rapidly and its implication for patients and biopharma companies and investors. For us in Pakistan, most of this may sound completely foreign, but we are already using many of these products. New Modalities L...

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...

New Gene Therapy Products – Part 1 – Asrar Qureshi’s Blog Post #746 Dear Colleagues!  This is Asrar Qureshi’s Blog Post #746 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: Ivan Samkov Photo Credit: Google Images 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. In this treatment, a gene can be delivered to a cell using a carrier called ‘vector’. Mostly, viruses are used as vectors due to their innate ability ...

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!  This is Pharma Veterans Blog Post #218. Pharma Veterans shares the wealth of knowledge and wisdom of Veterans for the benefit of Pharma Community. 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 . New Gene Therapy for SMA – ZOLGENSMA by Novartis FDA approved 59 NMEs (New Molecular Entities) in 2018 and 12 up to now in 2019. 2 , 3 . A review of these approvals sheds light on what is happening in the Drug Development Scene. Drug Development has changed in several ways in recent times. It is mostly going away from mainstream indications and towards specific ‘unmet needs’, rare diseases, orphan drugs, new classes such as Nibs (small molecule kinase inhibitors), Nabs (nanoparticle, Albumin based), Mibs (proteasome inhibitors), mAbs (monoclonal Antibodies based) and so on. The bot...