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After announcing plans to explore strategic alternatives less than a month ago, Genocea Biosciences is shutting down operations.

Imugene and City of Hope announced this week that the first patient has begun treatment in a revolutionary Phase I clinical trial, investigating the safety and efficacy of CF33-hNIS (Vaxinia), a cancer-killing virus, in adults with metastatic or advanced tumors. 

Regeneron Pharmaceuticals shared positive results from the company’s Phase III trial of a candidate drug for children with homozygous familial hypercholesterolemia (HoFH), a rare, life-threatening condition characterized by elevated circulating levels of low-density lipoprotein cholesterol (LDL-C) and accelerated, premature atherosclerotic cardiovascular disease (ACVD.

Biohaven Pharmaceutical said on May 23 the company’s experimental drug for patients with spinocerebellar ataxia, a genetic disease that affects the nervous system, failed to meet the main goal of a late-stage study.

Ocugen Inc. said on May 23 the U.S. drug regulator has lifted the clinical hold on a mid-to-late stage trial of the COVID-19 vaccine being developed by the company’s Indian partner Bharat Biotech.

AbbVie submitted a New Drug Application to the U.S. Food and Drug Administration for ABBV-951 (foscarbidopa/foslevodopa) for the treatment of motor fluctuations in patients with advanced Parkinson’s disease.

Diversity in all clinical research—including rare disease research—is currently under scrutiny, partly because of disparities experienced with COVID-19. Rightly so, as data show that 86 percent of genomics studies to date have involved people of European ancestry, which has limited the potential benefits of genomic research for many populations. Rebecca Sutphen, Co-Founder and Chief Medical Officer of InformedDNA, and Board-Certified Genetic Counselor Karmen Trzupek analyze strategies to increase diversity in rare genetic disease trials.

There was a significant drop-off in NME approvals by FDA in 2016, but there were not enough new drug applications with user fee goals to reach the lofty 2015 total.

Provides identification of potential patient safety issues and actionable insight into clinical trial site performance RESEARCH TRIANGLE PARK, N.C. – June 15, 2015 – Quintiles, the world’s largest biopharmaceutical services company, today announced the addition of advanced statistical monitoring and predictive analytics capabilities to its risk-based monitoring (RBM) solution. Predictive and Advanced Analytics combine Quintiles’ […]