General Injectables

Gene therapy has been a significant development in medicine for nearly 40 years, focusing on treating genetic disorders by injecting specific genetic material into a patient to alter cell activity and address hereditary illnesses. Four fundamental gene therapy techniques include gene silencing, gene addition, gene replacement, and gene editing. Adeno-associated viruses (AAVs) are a type of gene therapy vehicle that was initially discovered as a contaminant of adenovirus preparations. The first human gene therapy study was conducted in 1970 when American physician Stanfield Rogers tried to cure argininemia with a papillomavirus-containing arginase. The primary vector for in vivo gene therapy delivery is recombinant AAVs (rAAVs). The first rAAV gene therapy treatment, Alipogene tiparvovec (Glybera), received approval in 2012 for lipoprotein lipase deficiency. In 2017, the US FDA approved Luxturna (Voretigene Neparvovec) as the first retinal gene therapy for human use, specifically for patients with LCA type 2, an inherited retinal degeneration caused by mutations in the RPE65 gene. AveXis developed Onasemnogene abeparvovec (Zolgensma®) for spinal muscular atrophy (SMA). This blog summarises the emergence and applications of AAV in gene therapy platform. Read More

In this blog, our focus would be on autosomal dominant disorders and autosomal recessive disorders that can be cured with a gene therapy approach. Gene therapy has faced numerous obstacles and it took an extensive period of time for reaching up to the clinic from the research lab. However, continuous and rapid advancement in the molecular biology and genomics field set the stage to develop gene therapies for a range of inherited disorders. Because of the certain limitation of the application of drug and surgical treatment, some of the cardiovascular disease also needed gene therapy approaches. Though huge progress has been observed in the treatment of autosomal recessive disorders by delivering the normal exogenous genes that can restore the proper biological function of the affected or mutated gene. However, a similar outcome cannot be expected in the case of autosomal dominant disorder as precise differentiation is required between diseases/mutated alleles from that of healthy/unaffected alleles. Read More

Quite often we hear people talking about microbiome disturbance leading to unhealthy aging and going back to our ancestral habits including paleo diet has the potential to cure many diseases. This folk wisdom is supported by some recent scientific publications. However, we have majorly neglected the fact that paleo diet-eating and cave-dwelling ancestors of ours had several insects on their bodies, and inside caves, they constantly fought with insects. Thus we argue, if modern habits are responsible for the current epidemic of metabolic/cardiovascular/neurological and other degenerative diseases, may be insects also had some role to play in the healthier aging of our ancestors as compared to us. In this blog post, we would like to review the benefits of insect bitings/stings published in the literature. Thus we will examine, if an apparent parasitic interaction between humans and insects is a mutualistic relationship in disguise. When an insect bites/stings us, it releases a barrage of biologically active compounds, including those with potential to act as anticoagulant/vasodilator. Can these chemicals be exploited to cure Cardio-Vascular-Diseases/dissolve internal blood clots? More importantly, there are other chemicals which have virucidal, anti-cancer and antimicrobial properties, which in either native or modified form can be repurposed for pharmaceutical applications. Read More

Moving ahead with our blog series we are bringing up Base Editing a new feather in the cap of gene editing therapy. Base Editing Therapy is a technology that introduces single-nucleotide variants (SNVs) precisely and efficiently at targeted genomic sequences without causing double-stranded breaks in the DNA enabling it as an efficient technique of genome editing. Nearly half of known pathogenic genetic variants are due to SNVs and base editing therapy holds enormous potential for the treatment of these genetic disorders by either temporary RNA or permanent DNA base alterations. Correction of single point mutations will be a major point of interest in the upcoming times for the scientific community for precision medicine. Read More

The Indian pharmaceutical industry has seen an exponential growth in the field of fill finished dosage forms, especially generics but the future lies beyond generics in the field of complex generics, biosimilairs, vaccines and New Chemical Entities (NCE)/New Biological Entities (NBE). Developing NCEs and NBEs will position Indian companies in the ivy league of global innovators. Risk adverseness, lack of perseverance and complex, long regulatory approval process are impeding Indian pharma companies to venture into NCE/NBE research. Product portfolio expansion into complex generic injectables is an attractive high-return alternative for the Indian generic pharmaceutical industries. Read More