Real World Evidence

BBCR Company News

BBCR’s team of industry experts help our clients match treatments to rare genetic conditions and unsolved diseases, then work with a product developer on the best plan to market.

June 8th, 2021 | Real World Evidence

Drug repurposing acts to lower the need for early stage clinical trials and can help identify new uses for existing drugs. People tend to believe that a repurposed therapy can never be truly novel or transformative. Nothing could be further from the truth. One attractive option of Drug Repurposing is to use a scientific approach […]

Boston Biotech Clinical Research uses innovative approaches to de-risk your product development. For our clients interested in Proof of Mechanism and Proof of Concept – PoM and PoC – BBCR has the expertise to ensure successful product development at any stage of development.

June 3rd, 2021 | Real World Evidence
PoM and PoC Services Boston Biotech Clinical Research

The BBCR team designs Proof of Concept (PoC) Trials and Proof of Mechanism (PoM) studies with the drug clinical plan and regulatory strategy in mind. 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 […]

Application of CAR Cell Therapy in Solid Cancer

June 1st, 2021 | Real World Evidence

From CAR T to CAR macrophage: The improvement of CAR cell therapy in solid cancer treatment The Chimeric Antigen Receptor T (CAR T) cell technology is a revolutionary therapy and has shown promising clinical response in cancer treatment. In 2017, anti-CD19 CAR T cell therapy against B cell malignancies was approved by US FDA. However, […]

Electrographic Seizures and COVID-19

May 25th, 2021 | Real World Evidence

Electrographic seizures and other epileptiform patterns are common in patients with COVID-19 and associated with adverse outcomes By: Dr. Maria Niu There have been 137,866,311 confirmed cases of COVID-19, including 2,965,707 deaths in the world, according to the WHO reports of April 15, 2021(https://covid19.who.int). In addition to the typical pulmonary complication, neurological manifestations were also […]

Obesity Associated Gut Microbiota Alterations Worsen GVHD

May 19th, 2021 | Real World Evidence

Obesity-induced gut microbiome composition is linked with graft-versus-host disease (GVHD) in mice and human after allogeneic hematopoietic stem cell transplantation (HSCT) By: Dr. Maria Niu GVHD is a potentially severe immune disorder related to HSCT. In brief, the donor T cells recognize the recipient as non-self or foreign, therefore trigger a wide range of immune […]

Recent Advance of Gene Therapy in Rare Diseases

May 13th, 2021 | Real World Evidence

Gene Therapy improved vision in patients with leber hereditary optic neuropathy (LHON) By: Dr. Maria Niu Mitochondria, powerhouses of eukaryotic cells, is a certain kind of cytoplasmic organelle and plays a critical role in energy production. Mitochondria dysfunction results in a broad spectrum of multisystem disorders. LHON is a mitochondrial neurodegenerative disease typically caused by […]

New Drug Approved in the Field of SiRNA-based Therapeutics Targeting Rare Disease

May 5th, 2021 | Real World Evidence

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 […]

COVID-19 Pandemic and Mental Health

April 29th, 2021 | Real World Evidence

The Lancet: 34% COVID-19 survivors have neurological and psychiatric disorders By: Dr. Maria Niu Previous case reports had described the altered mental problems and psychiatric disorders in people diagnosed with COVID-19 infection. In the latest study (https://doi.org/10.1016/S2215-0366(21)00084-5) reported by The Lancet Psychiatry on April 6, 2021, the authors confirmed a high incidence of neurological and […]

The Effect of Fecal Transplant on Melanoma

April 28th, 2021 | Real World Evidence

Fecal microbiota transplantation (FMT) promotes the response of check point inhibitors (CPI) to melanoma patients By: Dr. Maria Niu Fecal microbiota transplantation (FMT) is a procedure that delivers fecal bacteria and other microbes from a healthy individual into another individual with various diseases or disorders. It has been recently tested in infection, immune conditions, and […]

Drug Development for Rare Duchenne Muscular Dystrophy (DMD)

April 20th, 2021 | Real World Evidence

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 […]