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Foundation Fighting Blindness and 4D Molecular Therapeutics Partner to Boost Retinal Gene Therapy Development

Promising gene therapies for inherited retinal diseases (IRDs) — many of which have moved into clinical trials — use viral delivery systems (aka vectors) to get copies of the therapeutic gene to the retinal cells that need them. Viruses are well suited when used for gene delivery, because they’re adept at penetrating cells with their genetic cargo. While some viruses in their natural state can cause illness, therapy developers remove the native genes and replace them with those that are therapeutic.

The Foundation Fighting Blindness and 4D Molecular Therapeutics (4DMT), an innovative leader in viral vector development, have formed a partnership to help companies and researchers quickly obtain and implement high-quality vectors for their retinal gene-therapy development efforts.

4DMT is an ideal partner for FFB, because the company specializes in development of adeno-associated viruses (AAVs), which are commonly used in emerging gene therapies for IRDs. Furthermore, 4DMT’s vectors can be customized to meet the requirements for treating a wide range of retinal conditions and are easily transferable to the clinic. The vectors will be designed for injection into the vitreous gel in the middle of the eye and to target specific types of retinal cells (e.g., photoreceptors, retinal pigment epithelium, etc.).

With its established connections with most retinal disease research labs around the world, the Foundation will serve as a matchmaker, helping scientists learn about and obtain the optimal vector technology for their projects.

“We are very impressed with 4DMT’s vector evolution approach, the company’s product pipeline, and manufacturing expertise. The potential is great for developing a number of gene therapeutics that could treat those affected by retinitis pigmentosa and allied conditions using a simple intravitreal injection approach,” says Patricia Zilliox, PhD, FFB’s chief drug development officer. “We are extremely excited by 4DMT’s collaboration with FFB, a globally recognized leader in the effort to cure blindness due to inherited retinal degenerations.”

“FFB has tremendous expertise identifying the best retinal research and an outstanding network of funded investigators and companies with whom we hope to collaborate to develop a portfolio of products that will benefit those affected with retinal degenerative diseases,” says David Kirn, MD, co-founder and chief executive officer of 4DMT.

4DMT has vector licensing partnerships with the following companies: Applied Genetic Technologies Corporation (AGTC), Pfizer, Roche, Benitec, and UniQure.

8 Responses to 'Foundation Fighting Blindness and 4D Molecular Therapeutics Partner to Boost Retinal Gene Therapy Development'

  1. Jeri Wesley says:

    Is there any progress in finding a cure for Retnitus pigmentosa?

  2. Jaye Anker says:

    My beautiful smart grandson has ushers, was born deaf and have 2 cochlear implants. Just found out about the usher, as he is soon to have a baby brother through genetic testing. Can u help, he sees alright now, except does not like the dark. Should we save the new baby cord blood

    • EyeOnTheCure says:

      Hi Jaye,

      It is important for your grandson to get genetically tested and potentially his new brother. Talk to a genetic counselor about ramifications (pros and cons) of testing. Cord blood will likely not be helpful. Keep in mind that many emerging RP treatments will help people with Usher, because Usher is RP with hearing loss.

  3. Larry Heath says:

    This research sounds promising and would be of great benefit to me. I have RP and have had it since birth. I am now 61 years old and have limited sight as the RP has progressively gotten worse over the years. I would like more information and possibly a way to get on a list for this treatment.

  4. Alan Shively says:

    Alan has Ushers and is blind. He has a cochlear cochlear implant and a hearing aid. We want more information about your treatments

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