Drug Discovery World

NOVEMBER 28, 2023

Importance of healthy mitochondrial function Dysfunctional mitochondria are usually tagged for removal via mitophagy with a protein ‘flag’ called ubiquitin. This paper strongly supports further study of USP30 inhibition as a potential disease-modifying therapy for PD.”

In-Vivo 52

In-Vivo Protein Drugs Research 52

XTalks

AUGUST 19, 2022

The cells are prepared outside of the body ( ex vivo ) and then infused into the patient. Zynteglo’s hefty price tag of $2.8 Although the therapy is administered to the patient once, the treatment process has several steps that may take place over several months.

Gene Therapy 95

Gene Therapy FDA Approval Gene Clinical Trials 95

XTalks

DECEMBER 13, 2023

This approval also marks bluebird’s third ex vivo gene therapy approved by the FDA for a rare genetic disease and second FDA approval for an inherited hemoglobin disorder, cementing our position as a gene therapy leader.” Vertex-CRISPR’s Casgevy has a US list price of $2.2 million and bluebird’s Lyfgenia is 40 percent higher at $3.1

Gene Therapy 98

Gene Therapy FDA Approval Gene In-Vivo 98

Delveinsight

FEBRUARY 25, 2021

Electroporation is used for ex vivo delivery of therapies to blood and immune cells. The company’s in vivo therapies reach the eye and central nervous system via adeno-associated viruses, while its liver therapies employ lipid nanoparticles (LNPs). Beam employs three approaches to deliver its genetic medicines to cells.

DNA 52

DNA In-Vivo Genetics Medicine 52

Drug Discovery World

JUNE 14, 2023

We have now our lead product in a Phase I/II study, which is a HER-2 directed tag T cell product for patients with HER-2 positive solid tumours. There’s a huge translational gap going from drug discovery to our in vivo mouse models, and then on to a human subject. I’ve been with a company for more than five years now.

Research 52

Research Engineer In-Vivo Production 52

XTalks

FEBRUARY 27, 2024

Addressing the high price tag, Iovance’s commercial chief Jim Ziegler said, “Payers have expressed their appreciation for the value proposition for Amtagvi.” Vogt said Iovance set Amtagvi’s price based on its value as the first drug approved in the post-PD-1 melanoma setting, and the company’s analysis of relevant benchmarks.

FDA Approval 52

FDA Approval Manufacturing In-Vivo Trials 52

XTalks

AUGUST 17, 2020

This typically involves taking T cells from the body, engineering chimeric receptor antigens (CAR) and inserting them into the cells, expanding the cells ex vivo and injecting them back into the body. Why not use a biologic platform to essentially do in vivo cell therapy directly in the patient?”. “The

Protein 98

Protein Engineer Immune Response In-Vivo 98