Pink Sheet is part of Informa PLC

This site is operated by a business or businesses owned by Informa PLC and all copyright resides with them. Informa PLC’s registered office is 5 Howick Place, London SW1P 1WG. Registered in England and Wales. Number 8860726.

This copy is for your personal, non-commercial use. For high-quality copies or electronic reprints for distribution to colleagues or customers, please call +44 (0) 20 3377 3183

Printed By

UsernamePublicRestriction
UsernamePublicRestriction

How To Build An Endpoint: Spark Designed Testing Maze To Study Rare Vision Loss

Executive Summary

In an interview, Spark President Katherine High explains that multi-luminance mobility test used in clinical development of Spark's gene therapy voretigene sprang from 2011 FDA meeting on need to study functional vision in patients with retinal degenerative diseases. Experience highlights need for new endpoints when targeting previously untreated diseases, she says.

You may also be interested in...



The Luxturna Model: Spark’s Approach To Retinal Disease Endpoint Embraced In Gene Therapy Guidance

Established efficacy endpoints may not be suited to assessing gene therapies’ clinically meaningful effects in some rare retinal disorders, US FDA says in draft guidance that gives a shout-out to Spark’s development of a functional vision endpoint for voretigene; guidance also includes recommendations on appropriate control groups and use of sham injections.

Gene Therapy Manufacturing Remains Large Hurdle For Development, US FDA's CBER Director Says

Scale-up in particular is a challenge and FDA wants to help lower the barrier, CBER Director Peter Marks tells rare disease conference.

RMAT Designation, Gene Therapies Highlight 2017 CBER Transformation

CBER Director Peter Marks says the center implemented a lot in a short amount of time for the RMAT pathway.

Related Content

Topics

Related Companies

UsernamePublicRestriction

Register

PS121767

Ask The Analyst

Please Note: You can also Click below Link for Ask the Analyst
Ask The Analyst

Your question has been successfully sent to the email address below and we will get back as soon as possible. my@email.address.

All fields are required.

Please make sure all fields are completed.

Please make sure you have filled out all fields

Please make sure you have filled out all fields

Please enter a valid e-mail address

Please enter a valid Phone Number

Ask your question to our analysts

Cancel