Boston Biotech Clinical Research (BBCR) works with biotech, pharmaceutical, and device companies to develop a clinical/regulatory roadmap consisting of simplified clinical programs, streamlined protocols, and cost-effective trials. Our Boston-based, integrated, boutique consulting team specializes in rare disease and orphan indications, and is dedicated to supporting pharmaceutical innovators and nurturing each product’s strengths.
We help to Identify areas of need or economic interest that can help companies find homes for treatments for rare diseases and precision medicine. Developing drugs for rare diseases and precision medicine indications can be rewarding, but navigating the challenges is not for the faint of heart. Expert guidance is essential in an area where […]
FDA guidance (March 2020 and updated July 2020) acknowledged that the impact of COVID-19 may require companies conducting clinical trials to consider virtual patient visits or put new processes in place regarding their current protocols. Since COVID 19 has changed many of our normal way to conduct Clinical research, most organizations reported some level of […]
Pediatric Rare diseases and Rare Pediatric Disease Designation FDA will award priority review vouchers to sponsors of certain rare pediatric disease product applications that meet the criteria specified. Section 529 of the FD&C Act is intended to encourage development of new drug and biological products for the prevention and treatment of rare pediatric diseases. Section […]
Scientists launched studies in search of genes that could explain why some people infected with SARS-CoV-2 get really sick, while others have only mild symptoms. We know that chronic health conditions—such as hypertension and diabetes can play a role, but there are also evidences that people’s genes can influence how their bodies react to other […]
The gut microbiome serves many useful functions in the body, but it can also rev up the immune system in harmful ways. Zit has been pustulated that Diet can influence the microbiome and the mucosal immune response. In the paper: “Diet modulates colonic T cell responses by regulating the expression of a Bacteroides thetaiotaomicron antigen,” Sci Immunol, 4:eaau9079, […]
Today, it is widely accepted that NETs have both a protective and a pathological impact on the host. When neutrophils encounter pathogens, not only engage in phagocytosis and degranulation, they also release neutrophil extracellular traps (NETs) In CANCER, NET-associated proteins lead to reawakening of dormant cancer cells and convert them to proliferating metastatic cells. In […]
Gene therapies for disorders such as X-linked Severe Combined Immunodeficiency (SCID, sometimes known as “bubble boy disease”), and Spinal Muscular Atrophy (SMA) have, for the first time, shown remarkable safety and efficacy results in clinical trials. FDA approved Trikafta, a gene-based therapy creating hope for children with cystic fibrosis and Zolgensma, a gene therapy drug, […]
Autoimmunity occurs when the body is unable to differentiate “self” from “non-self” which results in overactive immune response against own cells and tissues. Autoimmune diseases affect 5 %-8% of the population; 78% affected are females. Low level autoimmunity is normal. Over 80 conditions linked to autoimmunity have been identified and 15 diseases directly linked to […]
Retrophin’s experimental PKAN drug fosmetpantotenate fails phase III trial Pantothenate kinase-associated neurodegeneration (PKAN) is a rare disease characterized by a progressive neurodegenerative disorder and buildup of iron in the brain which is estimated to affect up to 5000 patients worldwide. PKAN is inherited in an autosomal recessive manner and is caused by mutations in the […]