Drug Development

Biomarkers are now a routine part of drug development In rare diseases and precision medicine, implementation of biomarkers during product translation into clinic and early clinical development, moves treatment to market faster.

Given the global Covid19 pandemic and the global nature of the pharmaceutical and biotech industry, key operational areas have and will continue to be impacted. As with any emergency response, the key to management is implementing mitigation plans. Early stage pharmaceutical and biotech companies need to be proactive in developing contingency plans to jump start […]

Lumasiran, a siRNA Therapeutic drug for primary hyperoxaluria type1(PH1) By: Dr. Maria Niu PH1 is a rare genetic disease caused by deficiency of the liver peroxisomal enzyme alanine:glyoxylate-aminotransferase (AGT), resulting in overproduction of oxalate. The clinical manifestation includes kidney stones, widespread organ damage, and kidney failure. Liver transplantation is applied in PH1 patients to provide the […]

Progress and limitation of therapeutic approaches targeting DMD By: Dr. Maria Niu Duchenne muscular dystrophy (DMD) is an X—a chromosome-linked recessive disorder caused by mutations in the gene coding muscle cytoskeletal protein dystrophin. Because of the X-linked inheritance, nearly 1 in 3,500 males are affected worldwide, while females occasionally develop symptoms. There is no curative […]

Neuroblastoma is a childhood cancer that develops when immature nerve cells fail to develop into normal mature nerve cells. These tumors can begin anywhere in the body, but are often found in the abdomen or chest, neck, hips, and bone marrow. Despite intensive chemotherapy, neuroblastoma can be difficult to cure and the prognosis is often […]

Moderna published (https://medcitynews.com/2020/09/moderna-publishes-phase-iii-covid-19-vaccine-study-protocol-as-trial-enrolls-more-than-80-of-participants/?rf=1) that the Phase III Covid-19 vaccine study protocol enrolled 25,296 of its planned 30,000 participants which is more than 80% of participants. Of all enrolled subjects, 10,025 have received the full vaccine. This mRNA-1273 Moderna’s vaccine consists of two shots – one delivered on day 1, and a booster shot that participants […]

Pfizer, a pharmaceutical company, in partnership with BioNTech has reported that its lead vaccine candidates has shown promising results in protecting monkeys against coronavirus infections. Pfizer ‘s experimental vaccine is different from other vaccine candidates. This is in an mRNA format, which should allow for a better migration of the viral gene into the cell […]

A Priority Review designation does not alter the scientific/medical standard for approval or the quality of evidence necessary and it does not affect the length of the clinical trial period. Prior to approval, each drug marketed in the United States must go through a detailed FDA review process. FDA drug review time includes two-tiered system […]

The IND requires very detailed product and development data such as information related to manufacturing, data from nonclinical studies, and previous clinical studies related to the IMP. IND also requires a comprehensive source of documentation including study reports. It follows the CTD structure developed by ICH and is mandatory to be submitted in the eCTD […]

Techniques that bypass or trick the guardian of the central nervous system. Developing drugs that can penetrate into the brain requires some creativity. The approaches vary, each technique comes with its benefits and drawbacks, making it appropriate for some patients or drug types but not others. In some diseases, you might want the drug everywhere; […]