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Posts tagged clinical trials

Watch VISIONS 2018 LIVE on June 22 & 23

Get the inside scoop on this year’s top research advances and what’s next in retinal disease science by watching VISIONS 2018 LIVE!

We will be streaming several of our annual VISIONS conference sessions live on Friday, June 22 and Saturday, June 23. The sessions will be live streamed here on our blog, and our national Facebook as well.

Here’s the full schedule of live sessions:

Friday, June 22, 2018

  • 9:00 a.m. PDT – 10:15 a.m. PDT– Opening Session: Mission Possible! The Year’s Top Advances and What’s Next in Retinal Disease Science
  • 12:30 p.m. PDT – 2:00 p.m. PDT – Keynote Luncheon: Think Outside our Perceived Limitations
  • 3:10 p.m. PDT – 4:15 p.m. PDT – What You Need to Know About Genetics, Genetic Testing, and Patient Registries
  • 4:20 p.m. PDT – 5:25 p.m. PDT – Clinical Trials Update and Is a Clinical Trial Right for You?

Saturday, June 23, 2018

  • 9:00 a.m. PDT – 10:25 a.m. PDT – Retinitis Pigmentosa (RP) Research and Clinical Updates
  • 10:30 a.m. PDT – 11:40 a.m. PDT– Stress Management and Self-Care for People with Degenerative Diseases
  • 1:00 p.m. PDT – 2:35 p.m. PDT – Adapting and Thriving: Find Your Product – Tips and Tools for Peak Personal Performance
  • 3:05 p.m. PDT – 4:30 p.m. PDT – Closing Session: Featuring Biotech Executive Emmett Cunningham and Chapter Awards

The full list of the many sessions that will be live on the blog, with additional details, is also on our website here.

We hope you’ll tune in, share it with your friends, and learn more about the promising new advances in retinal research.

 

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ARVO 2018: Dr. Henry Klassen Provides Update on jCyte Stem Cell Trials

Dr. Henry Klassen, jCyte co-founder and investigator at UC Irvine, provides an update in the video below on the clinical trials for an RP therapy derived from stem cells.

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Top Retinal Research Advances for 2017

To view and listen to Ben Shaberman’s presentation of “Top Retinal Research Advances for 2017,” with full slides and audio, click here. The text to the presentation is as follows:

This is Ben Shaberman, director of science communications, at the Foundation Fighting Blindness (FFB), and I’m pleased to present a quick overview of some of the exciting research advances for inherited retinal diseases made during 2017. It has been an exciting year with several promising therapies moving into and through clinical trials.
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Stem-Cell Therapy Clinics Remain Inadequately Regulated, Pose Risk to Patients

Though the U.S. Food & Drug Administration (FDA) is more tightly regulating U.S. clinics that tout stem-cell trials and treatments for inherited retinal diseases (IRDs) and a host of other conditions, many significant loopholes and enforcement limitations remain.

The bottom line is: The FDA has not yet established regulations to adequately protect patients with IRDs seeking treatments from these clinics. If a clinic is charging for a stem-cell treatment or procedure for an IRD, it is probably not legit. The expense to the patient is a major red flag.
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FFB’s Investments Are Filling the Pipeline for Vision-Saving Therapies

GXM_7140With five gene-therapy clinical trials underway or soon to begin, Applied Genetic Technologies Corporation (AGTC) is generating tremendous excitement for the potential to overcome vision loss from several inherited retinal diseases.

At the Foundation’s Investing in Cures Summit on September 16 in Chicago, Sue Washer, AGTC’s chief executive officer, emphasized FFB’s crucial role in moving the company and its projects forward. “We as an organization would not be here today without FFB,” she said. “And that all started with the work that was funded by the Foundation in Bill Hauswirth’s lab at the University of Florida.” Bill Hauswirth, PhD, is one of AGTC’s scientific co-founders, and his groundbreaking gene-therapy research has been funded by FFB for 20 years.
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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.
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FFB-Funded Scientists Report on Nine Promising Translational Research Efforts

Translational research — moving promising science out of laboratories and into clinical trials — is essential to getting vision-saving, retinal-disease treatments out to the millions who need them. With that said, translational research is also costly and high risk and requires extensive clinical development and regulatory knowledge.

The Foundation Fighting Blindness has taken the translational challenge head on by investing more than $75 million in therapy-development projects with strong clinical-trial potential through its Translational Research Acceleration Program (TRAP), which includes Gund-Harrington Scholar Awards.
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ID Your IRD: A Free Genetic Testing Program for Eligible People with Inherited Retinal Diseases


Genes are like the blueprint or code for determining who we are. We all have about 23,000 pairs of genes in most cells in our bodies. Many of our physical attributes — such as height, eye and hair color, and complexion — are determined by our genes.

However, certain misspellings, also known as mutations, in our genetic code can cause diseases or increase our risk for them. In fact, inherited retinal diseases are caused by mutations in single genes.
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Unregulated Stem-Cell Therapy Causes Severe Vision Loss for Three Florida Women

A report today in The New England Journal of Medicine (NEJM) describes the cases of three women with age-related macular degeneration (AMD) who lost much of their eyesight after receiving ocular injections of stem cells derived from their own fat tissue. All of the women had good enough eyesight to drive before the procedures. Each paid $5,000 to receive the injections from a private clinic in Sunrise, Florida. The New York Times and other major media outlets have published news stories on the NEJM report.
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AGTC Leverages Funding from the Foundation to Move Promising Treatments into Clinical Trials

Seed becoming a plantCompany Builds on FFB’s Initial Investment to Garner $265 Million in Therapy Development Funding

In the early 1990s, scientists began discovering the genetic defects causing blinding, inherited retinal diseases and saw a unique opportunity to overcome them. They envisioned gene therapy — delivering healthy genes to the retina to replace the bad ones — as an elegant approach to saving and restoring vision. Furthermore, a single injection of gene therapy would likely halt or reverse the disease process and work effectively for several years, perhaps the patients’ lifetimes.

The Foundation Fighting Blindness, the world’s leading private, nonprofit retinal research organization, funded most of these genetic discoveries for retinal diseases and immediately recognized the enormous opportunity for gene therapy to beat blindness.
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