[NFBP-Jigsaw] Fwd: Fw: Partner News: Clinical Trial Recruitment for Patients with ABCA4-Related Stargardt Disease

Thu Jan 15 01:29:36 UTC 2026

Hello all:

Please see below for a research opportunity for those who have Stargardt
Syndrome.  I am not affiliated with this study so please contact the
appropriate people in the email below.

Thanks,

Stacie

Stacie Leap
President, Greater Philadelphia Chapter &
President, Pennsylvania Organization of Blind Parents (POBP),
National Federation of the Blind of Pennsylvania

 stacie.leap at gmail.com | 215-776-6741

The National Federation of the Blind advances the lives of its members and
all blind people in the United States. We know that blindness is not the
characteristic that defines you or your future. Every day we raise the
expectations of blind people, because low expectations create obstacles
between blind people and our dreams. Our collective power, determination,
and diversity achieve the aspirations of all blind people.

---------- Forwarded message ---------
From: Downing, Patrick <patdowning at pa.gov>
Date: Wed, Jan 14, 2026 at 6:40 PM
Subject: Fw: Partner News: Clinical Trial Recruitment for Patients with
ABCA4-Related Stargardt Disease
To: Stacie Leap <stacie.leap at gmail.com>

Hi Stacie,

Can you get this resource out below to more people?! Or especially the
members with you know with Stargardt disease.

Patrick Downing (he/him)| Vocational Rehabilitation Counselor, MSW

Labor & Industry | Office of Vocational Rehabilitation

Bureau of Blindness and Visual Services | Philadelphia

801 Market St., Suite 6034 | Philadelphia, PA 19107

Phone: 215.929.4364 | Fax: 215.413.0608

Office Hours: 8:00 AM – 4:00 PM

www.dli.pa.gov/ovr<http://www.dli.pa.gov/ovr>

________________________________
From: Kerrigan, Patricia <pkerrigan at pa.gov>
Sent: Wednesday, January 14, 2026 3:33 PM
To: LI-OVR-BBVS-Phila <li-ovr-bbvs-phila at pa.gov>
Subject: FYI: Partner News: Clinical Trial Recruitment for Patients with
ABCA4-Related Stargardt Disease

Good afternoon all, sharing FYI good news Re: Clinical Trials for
Stargardt's,

Maurie Kerrigan | COMS,CLVT [cid:image001.jpg at 01DC856B.27DC0DC0]

Orientation and Mobility Specialist

Low Vision Therapist

Phone: 215.268.2872

pkerrigan at pa.gov<mailto:pkerrigan at pa.gov>

Begin forwarded message:

From: Foundation Fighting Blindness <membership at fightingblindness.org
<mailto:membership at fightingblindness.org>>
Date: January 13, 2026 at 2:30:38 PM EST
Subject: Partner News: Clinical Trial Recruitment for Patients with
ABCA4-Related Stargardt Disease

[Foundation Fighting Blindness logo]<
http://www.fightingblindness.org/?vcrmeid=WruxaV2hzEGdYLKerc7bPg&vcrmiid=KhEoLCXiJEWEmQ5IbHwRCw
>

[Various dark blue beams with text in the middle that says,]

CELESTE Trial Now Open for Individuals with Stargardt Disease

The CELESTE study is a first-in-human Phase 1/2 interventional clinical
trial to address ABCA4-related Stargardt Disease. It is sponsored by
AAVantgarde Bio<
https://u35158275.ct.sendgrid.net/ls/click?upn=u001.TwpWPek5144WRusuAV3MCcSykhxYRq-2F3UTeJnGsLKEiYG1jzzi-2BdB4gWlv9Wrg0F8-2B79vgZ85L-2BQZtsk-2FpPRF2TMJpVMaRlnMJczLTszqpAYQdIF6-2BwuezrIZuLVJ9VMPc6Q2lvOWitwFjzNXBUhgw-3D-3DVWN6_R7JeeiVU-2FISa3llGJQwIAFBekgh9jbcsFXWG3RMbPmO1dhAlhORgKFBR7-2F3-2Fgq85UtsHkSxEfWugYRQduSWmbIsmigDd1g061qjc1fflC22NUbVY5wxzLpCrpIFTnlyFhoZrvQQqRucwYZ-2BgtmPIZy3dfulrHmLW69svspVSLn1BaP6xLE0igeEYJEL3XN96d-2FIBujqyK2FT41Wpl4Lqg-2BLlSE5gsRR6Vv-2BL3UgG9hvD2BpY2BqoAcliYHDY2rVpjNCiYIy2P7MOanpNRV0fyEH8gd9Q-2FjD64QsTgbffQZc-3D
>.

The purpose of this study is to investigate the safety and efficacy of an
investigational gene therapy called AAVB-039, which aims to slow down or
halt vision loss by addressing the genetic root cause of Stargardt disease,
due to variants in the ABCA4 gene.

The study leverages AAVantgarde’s dual AAV technology to deliver a
functional copy of the ABCA4 gene into the retinal cells via a one-time,
single subretinal injection.*

Where will the study take place?

The study will be carried out at several sites in the U.S., the U.K., and
Europe. All study visits, including eligibility screening, surgery, and
follow-up visits, will be completed at the site.

Find out if you’re eligible:

The study is recruiting participants who meet the defined inclusion
criteria, including (but not limited to):

  *   Male or female, 8-55 years of age.
  *   Confirmed genetic diagnosis of Stargardt disease due to variants in
the ABCA4 gene.
  *   No previous treatment with an investigational gene therapy.

There will also be additional assessments carried out at the study site to
make sure the study is suitable for you.

Discover how you can take part:

For more details about the study, please visit ClinicalTrials.gov<
https://u35158275.ct.sendgrid.net/ls/click?upn=u001.TwpWPek5144WRusuAV3MCd1zP7OHVTXvbNQGMYVx5OqeqqBP1P-2FtL2Ug15FzfceBrhw53dy8E9ZKPyJD54QXZKdHInLqoMchUtxVvq7p-2BcpZAoJ6vBRnHJ4XKyvRVfgDTasf5QNNRL8pJNJyPCE7e-2FTK0gJ8Wms-2F6KiIku5LHko-3Ddtrw_R7JeeiVU-2FISa3llGJQwIAFBekgh9jbcsFXWG3RMbPmO1dhAlhORgKFBR7-2F3-2Fgq85UtsHkSxEfWugYRQduSWmbIsmigDd1g061qjc1fflC22NUbVY5wxzLpCrpIFTnlyFhoZrvQQqRucwYZ-2BgtmPIZy3dfulrHmLW69svspVSLn2LzgME7DmY1R2SSt-2B9F1tPkEvNxHXAOVaotxrF6SrasLoIZVgGC7Kpe4Qu1Y6aStvJBDoKMVbtveSZA5GQA1BeMjDD-2BMzVNwc4kwdMw23xf9gPJkszk31Hda6IZVS6MkE-3D>,
or you can contact AAVantgarde at ClinicalTrials at Aavantgarde.com<mailto:
ClinicalTrials at aavantgarde.com
?vcrmeid=WruxaV2hzEGdYLKerc7bPg&vcrmiid=KhEoLCXiJEWEmQ5IbHwRCw>.

About Stargardt Disease

Stargardt disease is an inherited retinal dystrophy, most commonly caused
by variants in the ABCA4 gene, that can start from childhood or
adolescence. A protein that is needed for the normal function of
light-sensing cells in the back of the eye (retina) is not produced due to
an error in the ABCA4 gene. It is the most common inherited macular
degeneration, affecting approximately 1 in 8,000 to 10,000 people.1
Currently, there are no approved treatments for Stargardt disease.

About AAVantgarde Bio

AAVantgarde Bio (AAVantgarde) is a clinical-stage biotechnology company
advancing gene therapies for patients with inherited retinal diseases. The
company’s lead programs target Stargardt disease and retinitis pigmentosa
due to Usher syndrome type 1B, two severe inherited retinal diseases with
no approved treatments. With a strong foundation in translational science
and a commitment to clinical excellence, AAVantgarde is working to bring
transformative therapies to patients.

For more information, please visit the AAVantgarde website.<
https://u35158275.ct.sendgrid.net/ls/click?upn=u001.TwpWPek5144WRusuAV3MCcSykhxYRq-2F3UTeJnGsLKEjoMF4MMqxjGppjhYl0tcQW8sN-2F17Zhc1B7xOUpWVAy-2BKMg0gNA4B1XeSRwPZFTxuKP6UsJBi7HKkArIm32DON4sGGTG766iP77FUBl6XDiRN7QTXTPeqz6z7jaM0YUaP0-3DN08a_R7JeeiVU-2FISa3llGJQwIAFBekgh9jbcsFXWG3RMbPmO1dhAlhORgKFBR7-2F3-2Fgq85UtsHkSxEfWugYRQduSWmbIsmigDd1g061qjc1fflC22NUbVY5wxzLpCrpIFTnlyFhoZrvQQqRucwYZ-2BgtmPIZy3dfulrHmLW69svspVSLn1FFxeBdX-2F7yjlfrzlTf9Mrug3b2Oz39Ob31Dri07l0i58l4V7FSLmIVOXbtX2hlACHAzYcj-2B2JuZPtRupgu-2FbBuNANuR-2BZ4YhagAsIG7r4-2BJwPs3VcVIZ5j3qKXm2ar3k-3D
>

[LEARN MORE]<
https://u35158275.ct.sendgrid.net/ls/click?upn=u001.TwpWPek5144WRusuAV3MCcSykhxYRq-2F3UTeJnGsLKEjoMF4MMqxjGppjhYl0tcQW8sN-2F17Zhc1B7xOUpWVAy-2BKMg0gNA4B1XeSRwPZFTxuKP6UsJBi7HKkArIm32DON4sGGTG766iP77FUBl6XDiRN7QTXTPeqz6z7jaM0YUaP0-3D3k30_R7JeeiVU-2FISa3llGJQwIAFBekgh9jbcsFXWG3RMbPmO1dhAlhORgKFBR7-2F3-2Fgq85UtsHkSxEfWugYRQduSWmbIsmigDd1g061qjc1fflC22NUbVY5wxzLpCrpIFTnlyFhoZrvQQqRucwYZ-2BgtmPIZy3dfulrHmLW69svspVSLn3OV7wgL-2BG5EfAXXSXHeAjBUWC3AW5wLUBOhHPLk3GVeqTP2gAPgQQX6atAFL1tA4UZb0m9Iu0pUHnBi93-2FknSA56ZpgVbMx9Lr8pfMdWGxjvHL4Anf2QyYlZKwJSW9U-2BY-3D
>

*Please note, AAVB-039 is an investigational drug product whose safety and
efficacy has not been evaluated by any regulatory authority.

[DONATE]<
https://u35158275.ct.sendgrid.net/ls/click?upn=u001.TwpWPek5144WRusuAV3MCR-2BhknYyAyQYw4yUD-2Bqe9tHHr3HZKCjcw-2BzFfSB0ZYsyWrM3yASlky59myl39HwcFbT-2FQgueWlm-2BKHloVIp-2F-2FnSC3Ps-2Fcpjv4g6HOiFOe0wUZTqEFS-2FOeMjRGmJBvcQWwqYMfnAorgXGlDipd32IZWFKGnWdaQOFxE0mOdvt6swzvGwpZsl4NFuno1dQ-2BEK88g-3D-3D8m-C_R7JeeiVU-2FISa3llGJQwIAFBekgh9jbcsFXWG3RMbPmO1dhAlhORgKFBR7-2F3-2Fgq85UtsHkSxEfWugYRQduSWmbIsmigDd1g061qjc1fflC22NUbVY5wxzLpCrpIFTnlyFhoZrvQQqRucwYZ-2BgtmPIZy3dfulrHmLW69svspVSLn2o0HvIkkq-2FeyUYB94spfbVKqZNqAVE5ru9jDyQom0gt0bUeKmwfDrtaRNz6f4Qm87CeoD10mNBoNak79u-2BeMZRo44VDGb-2BeZ-2BPzm7-2FK75TCK1BgipJ-2FU3nh2y31WCoZSg-3D
>

© 2026 Foundation Fighting Blindness, All Rights Reserved.
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