article thumbnail

10 Key Learnings from Successful Cellular and Gene Therapy Trials for Rare Diseases

XTalks

Approximately 72 percent of rare diseases are genetic, and around 70 percent of rare genetic diseases emerge in childhood. This is important since not all pharmacies and sites are eligible to prepare and administer gene therapy due to biosafety and technical reasons. There needs to be a mock run done with sites and pharmacies.

article thumbnail

REGN-COV2 Independent Data Monitoring Committee Recommends Holding Enrollment in Hospitalized Patients with High Oxygen Requirements and Continuing Enrollment in Patients with Low or No Oxygen Requirements

The Pharma Data

To develop REGN-COV2, Regeneron scientists evaluated thousands of fully-human antibodies produced by the company’s VelocImmune ® mice, which have been genetically modified to have a human immune system, as well as antibodies identified from humans who have recovered from COVID-19.

Insiders

Sign Up for our Newsletter

This site is protected by reCAPTCHA and the Google Privacy Policy and Terms of Service apply.

article thumbnail

FDA Approves Zokinvy (lonafarnib) for Hutchinson-Gilford Progeria Syndrome and Processing-Deficient Progeroid Laminopathies

The Pharma Data

Progeria and Progeroid Laminopathies are separate and distinct ultra-rare, genetic, premature aging diseases that accelerate mortality in young patients. Eiger licensed exclusive worldwide rights to lonafarnib from Merck, known as MSD outside of the United States and Canada. ABOUT THE PROGERIA RESEARCH FOUNDATION.

article thumbnail

Regeneron’s COVID-19 Outpatient Trial Prospectively Demonstrates that REGN-COV2 Antibody Cocktail Significantly Reduced Virus Levels and Need for Further Medical Attention

The Pharma Data

To develop REGN-COV2, Regeneron scientists evaluated thousands of fully-human antibodies produced by the company’s VelocImmune ® mice, which have been genetically modified to have a human immune system, as well as antibodies identified from humans who have recovered from COVID-19.

article thumbnail

New England Journal of Medicine Publishes Positive Initial Regeneron Antibody Cocktail Results in Non-hospitalized Patients with COVID-19

The Pharma Data

To develop this novel medicine, Regeneron scientists evaluated thousands of fully-human antibodies produced by the company’s VelocImmune ® mice, which have been genetically modified to have a human immune system, as well as antibodies identified from humans who have recovered from COVID-19.

article thumbnail

Regeneron Announces Encouraging Initial Data from COVID-19 Antibody Cocktail Trial in Hospitalized Patients on Low-flow Oxygen

The Pharma Data

The EUA is temporary and does not take the place of a formal biologics license application (BLA) submission review and approval process and the use of the antibody cocktail remains investigational. Food and Drug Administration (FDA) in high-risk patients who have confirmed COVID-19 but are not currently hospitalized.

article thumbnail

A history of the pharmaceutical industry

pharmaphorum

The 21st century pharmaceutical and biotech sector has come a long way from its roots in 19th century pharmacy. Originating as a pharmacy founded in Darmstadt in 1668, it was in 1827 that Heinrich Emanuel Merck began the transition towards an industrial and scientific concern, by manufacturing and selling alkaloids.

Medicine 145