Neutrophils use an enzyme called neutrophil elastase (NE) to cleave bacteria. Human neutrophils release NE which looks like a fibrous structure like webs. These webs, able to trap bacteria, are called neutrophil extracellular traps (NETs). These webs are constituted of NE, other proteins, and copious amounts of DNA. It is important to keep in mind […]
We’ve had a few events where they’ve jumped from animals into people. There’s a lot being done on how coronaviruses infect people from animals. We know the real key is to know what the host cell receptor is—that’s the protein on the surface of cells that viruses bind to and invade. For SARS coronavirus, the […]
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, […]
Worldwide, over 4,000 patients with Gaucher disease have received enzyme replacement treatment (ERT), which is safe and well tolerated. Gaucher disease is a rare disease caused by mutations in GBA1. GBA1 mutations drive extensive accumulation of glucosylceramide (GC) in immune cells in the spleen, liver, lung and bone marrow. Extensive GC storage induces complement-activating IgG […]
BBCR Consulting, for the first time this year, decided to attend the European edition of the Gene Therapies in Rare Disorders conference in London UK. In our opinion, the event, as promised, proved to be: ”…uniquely focused conference that will bring the leaders in the field together to discuss the critical factors involved in driving […]
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 […]
A Clear Path to Approval The Strategic Clinical Innovation Organization (SCIO) concept developed by BBCR was designed specifically to help pharmaceutical innovators address the concerns and maneuver around evolving challenges. SCIO allows for time and cost efficiencies, and risk mitigation. The BBCR team is armed with extensive clinical, regulatory and industry experience that we use […]
Proof of Mechanism (PoM) Usually in Healthy Volunteers, Phase 1 study Essential for the selection of appropriate dose for PoC, disease model and biomarkers Investigate drug concentration at the target site of action Investigate drug engagement with target molecular receptor or enzyme (e.g. Receptor binding Investigate if drug biological response after target binding match the […]
Every clinical study has its unique challenges that initially may not have been factored for. Experienced management can help sponsors to address prolonged trial timeline and high quality data. Rare Diseases and Precision Medicine Require Unique Approaches In Clinical Trial Design. There could be any number of factors that could necessitate a study being rescued. […]
Our industry is looking for an innovative process. Focus must go to cost containment and value-based developments that allow sponsors to move more treatments to market faster. BBCR’s team expertise in rare diseases and precision medicine combined with SCIO approach helps streamline trials for swift regulatory approvals. BBCR specializes in the strategy and delivery of […]