Orphan Drug & Rare Disease Development: Understanding the European and US Regulatory Landscape

Premier Research

Today more than ever researchers are focused on providing care for diseases that in the past have remained neglected. The National Institute of Health (NIH) has identified around 7,000 rare diseases, many of which are of genetic origin.
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Spotlight

An associate professor of neurosurgery at the University of Pennsylvania, D. Kacy Cullen, is developing a new approach for treating patients that have neurodegenerative disorders like Alzheimer’s or Parkinson’s disease. Our brain is made up of a complex network of neurons. The paths that electrical impulses travel as they move through these neurons is what makes the brain work. But after injury or during disease, those pathways can break down. Retrieving them is not possible with current tools, and it's a major impediment to treating neural degeneration.


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Drug Safety Regulatory Requirements

DIA

This module is part of the DIA Drug Safety eLearning Program. The Drug Safety Regulatory Requirements module provides information about pharmacovigilance harmonization initiatives and regulatory requirements around world. This module includes harmonization initiatives, key US and EU regulations, the roles that ICH and CIOMS play in drug safety regulations, good clinical and pharmacovigilance practices, and standard operating procedures (SOPs) that support drug safety.
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Shifting the Cancer Vaccine Paradigm: NGS Best Practices for Developing Personalized Neoantigen Vaccines

Xtalks Webconferences

Precision medicine strategies have changed how the industry is approaching the development of cancer treatment vaccines, from targeting tumor-specific mutations to harnessing the power of the immune system.
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Optimizing Drug-Dose Checking to Minimize Alert Fatigue

Wolters Kluwer Clinical Drug Information

Drug dosing errors account for nearly 40% of all preventable medical errors and could prove harmful to patients and costly to healthcare organizations. Join MetroHealth System CMIO Dr. David Kaelber for a look at how his institution’s strategic approach to customizing its EHR-integrated drug dosing data lead to both improved patient outcomes and a reduction in irrelevant alerts and alert fatigue, as well as techniques for optimizing your drug decision support system.
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Cell and Gene Therapies for Cancer: Future Promises and Challenges

Bio-Techne

The field of immuno-oncology has exploded in the clinic, in the press, and on Wall Street, particularly regarding the use of genetically modified immune cells to fight cancer. This “explosion” is largely due to the success of chimeric antigen receptor (CAR) T-cell therapy. The fields of immunology and gene therapy have converged to harness recombinant viruses to deliver “living drugs” with remarkable clinical efficacy, especially for patients with blood cancers. Multiple approaches to engineering immune cells mostly T cells but also other immune cell types make use of CARs, while tumor-specific T-cell receptors continue to gain a foothold in the field, mostly for the treatment of solid tumors. Several genome-editing approaches are now available, including established technologies such as zinc-finger nucleases, and newer modalities like the CRISPR/Cas9 system. These methodologies have recently been applied to augment the antitumor efficacy of adoptively transferred cells in the clinic by knocking out negative regulatory molecules such as PD1.
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Spotlight

An associate professor of neurosurgery at the University of Pennsylvania, D. Kacy Cullen, is developing a new approach for treating patients that have neurodegenerative disorders like Alzheimer’s or Parkinson’s disease. Our brain is made up of a complex network of neurons. The paths that electrical impulses travel as they move through these neurons is what makes the brain work. But after injury or during disease, those pathways can break down. Retrieving them is not possible with current tools, and it's a major impediment to treating neural degeneration.

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