Dissatisfied with the limitations of AAV gene editing, Drs. Ray Tabibiazar and Joe Higgins launched SalioGen, a company with an ambitious goal: the safe delivery of large strands of DNA cargo that mimics the way it happens in nature. 

The manufacturing processes for biopharmaceuticals do not directly utilize fossil fuels, but fossil fuels are used in the generation of raw materials, consumables, and packaging and in supplying energy to building systems and indirectly to manufacturing equipment. This article looks at the key considerations you should make when considering decarbonizing your facility and uses a case study with quantification of estimated costs.

The pre-IND meeting takes place when a sponsor presents their planned Phase 1 study design, including their proposed product development plan and regulatory strategy, to the FDA. Think of this as a necessary milestone for a quicker development timeline. This article shares best practices for preparing for the meeting as well as for the meeting itself.

Safety risks can be described and modelled as cause-and-effect relationships using system risk structures. This article structures risks beginning with a defined danger or threat so they can be effectively understood and then managed. Examples include wearing protective gloves (as a simple risk) and handling antibody-drug conjugates (as a complex risk).

Survival rates have increased for many cancers over the past two decades, but there remains an unmet need for more effective therapies. This article examines the current state of clinical development, investment, and partnerships for novel immuno-oncology cell and gene therapies.

A complete model of the tumor biology is showing promise to become a new standard for predicting patient response and helping more patients benefit from the power of immunotherapy.

A new FDA guidance released in Dec. 2021 acknowledges the difficulties inherent in manufacturing individualized antisense oligonucleotide (ASO) treatments and offers researchers and manufacturers the opportunity to create policies and procedures that are more likely to be condoned by the FDA in an IND submission.

Outsourcing and technology have morphed our industry into a new decentralized model. Now, each sponsor must carefully develop an ongoing compliance strategy considering its unique business model and its influence on the drug development journey. Strategies are needed to design QMS' that can expand and pivot in tandem with corporate and clinical development.

In gene therapy development and manufacturing, developing and validating appropriate potency assays that reflect the mechanism of action acceptable to regulators is a process fraught with challenges. The FDA advocates the use of a matrix approach, and this article shares best practices of this approach.