Challenges and Solutions for Gene Therapy manufacturing

whitePaper | August 25, 2022

Patient engagement has become a measure of healthcare analytics that many in rehab therapy have sought to quantify.

whitePaper | December 15, 2022

Regulatory teams at life science companies are finding that effective regulatory information management (RIM) has become critical to their operations.

whitePaper | February 23, 2023

Phenylmethylsulfonyl fluoride, better known as PMSF, is a small molecule serine protease inhibitor used to prevent unwanted proteolysis.

whitePaper | January 24, 2022

The American Society of Gene & Cell Therapy (ASGCT) is the primary professional membership organization.

whitePaper | March 16, 2021

Cell and gene therapies have emerged as a new treatment paradigm, and with them has come the potential to alter the course of many rare diseases. In many cases, there is an opportunity to correct the underlying dysfunction with a one-time administration of a therapy and provide either a functional cure or a substantial improvement in health outcomes. In other cases, long term symptom relief will be the primary therapeutic objective. Some of these indications may require more than one dose to achieve continued symptom relief and scientists and companies are working on technologies that will enable the use of a repeat dose.

whitePaper | May 10, 2022

This guidance describes the benefit-risk principles applied by FDA when conducting product quality-related assessments of chemistry, manufacturing, and controls (CMC)2 19 information 20 submitted for FDA assessment as part of original new drug applications (NDAs) under section