Clinical Trials

ABO-102, Phase I/II Clinical Trial
Study: Phase I/II Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH
Indication: MPS IIIA (Sanfilippo A Syndrome)
Study Type: Interventional
Status: Active
Additional Information:

EB-101, Phase I/II Clinical Trial
Study:Phase I/II Gene Transfer Clinical Trial of NCT01263379
Indication: RDEB (Epidermolysis Bullosa Dystrophica)
Study Type: Interventional
Status: Active, This study is currently recruiting participants
Additional Information:

ABO-101, Phase I/II Clinical Trial
Study:Phase I/II Gene Transfer Clinical Trial of rAAV9.CMV.NAGLU
Indication: MPS IIIB (Sanfilippo B Syndrome)
Study Type: Interventional
Status: Active
Additional Information:

Pre-Approval Access Policy

Patient Focused

Developing rare disease therapies requires new approaches and collaboration between researchers, industry, regulators and patient groups. Abeona Therapeutics was forged from our close collaborations with key stakeholders who include:

Foundations + Patient Advocacy Groups

United States

Abby Grace Foundation

The Abby Grace Foundation remains HOPEFUL for a CURE. There is exciting research on the horizon for children affected with Sanfilippo Syndrome. Gene Therapy has shown promising results in animal models is pending the start of a Clinical trial at Nationwide Children’s Hospital in Ohio.

Ben's Dream

Ben’s Dream – the Sanfilippo Research Foundation, Inc. is a public 501(c)(3) non-profit organization that was formed in celebration of Ben’s 5th birthday. Its mission is to raise awareness of Sanfilippo Syndrome and sponsor research toward a cure. 

Jennifer and Stuart made the decision to form Ben’s Dream after working cooperatively for two years with the Children’s Medical Research Foundation (CMRF), another afflicted family’s foundation in Illinois. In January 2000 Stuart applied for and was granted a one year leave of absence from Xerox Corporation as part of the company’s Social Service Leave program to expand CMRF’s fundraising efforts. Believed to be the oldest program of its kind in American business, the leave afforded Stuart the opportunity to develop programs, raise funds and build the framework for Ben’s Dream.

The Children's Medical Research Foundation

Twenty-three-year old Kirby Wilson was diagnosed with Sanfilippo “B” Syndrome, a rare and catastrophic genetic disorder in 1995, at the age of four. Kirby is one of the thousands of afflicted children worldwide. There is no cure or treatment…yet.

The Children’s Medical Research Foundation, Inc, was formed by Kirby’s parents in 1995 to fund medical research and find a cure for Sanfilippo Syndrome and other neuro-genetic disorders. To date, the not-for-profit Foundation has granted over $3.7 million to eight research groups with more than 95% of all receipts appropriated to fund research. We invite you to learn more about Sanfilippo Syndrome, the Foundation, its work and how to help pave the way for healthy future for Kirby and others like her.

National MPS Society

The National MPS Society exists to find cures for MPS and related diseases. We provide hope and support for affected individuals and their families through research, advocacy and awareness of these devastating disorders.

Cure Sanfilippo Foundation

Cure Sanfilippo Foundation is a 501c3 non-profit organization whose mission is to advocate for and fund research directed towards a cure or treatment options for patients with Sanfilippo Syndrome. The Foundation was formed in 2013 by the parents of Eliza O’Neill, who was diagnosed with Sanfilippo at age 3.

Cure Sanfilippo Foundation supports the most promising scientific research around the world. We also increase awareness among medical professionals, media, and the general public, in the goal to help families and children receive an earlier diagnosis. Due to the rapid degeneration of this disease, it is literally a RACE to help Sanfilippo children. Today there is no proven cure or treatment, but we are out to change that.


Debra of America , The Dystrophic Epidermolysis Bullosa Research Association of America, is the only U.S. nonprofit providing all-inclusive support to the EB Community, through funding research for a cure and by providing free programs and services for those with: Epidermolysis Bullosa – “The Worst Disease You’ve Never Heard of.”

The mission of debra of America is to improve the quality of life for all people living in the United States with EB, their families, and caregivers through free programs and services while funding research to find a cure and treatments for Epidermolysis Bullosa.

EB Medical Research Foundation

The Epidermolysis Bullosa Medical Research Foundation (EBRF) is dedicated to finding a cure for Epidermolysis Bullosa by funding the best of cutting edge and innovative research and seeking out new researchers and scientists at America’s top institutions.

We have made great strides over the years in developments of new treatments, but our fight is far from over; it’s why we are urgently working on all fronts to support and fund these breakthroughs in research as a means of accelerating the delivery of these treatments to patients and to the marketplace. Through our ongoing funding of individual research programs at acclaimed Universities, through our funding of the EBCRC and our annual fundraisers, we are leading the way in this fight. We spend research dollars efficiently, harnessing our efforts and joining forces with others in the field.

Taylor's Tale

Taylor’s Tale is one of the world’s leading voices in the fight against rare disease. Founded in part to save one little girl with Batten disease, today we work to build a better future for one in 10 Americans – and 350 million people worldwide – who suffer from one of more than 7,000 rare diseases.

In many cases, rare disorders have devastating consequences and zero approved treatments. By supporting patients and their families, advancing breakthrough treatments and advocating for life-changing legislation, we are continuing Taylor’s powerful legacy.

Team Sanfilippo Foundation

Team Sanfilippo Foundation is a non-profit medical research foundation started by a group of parents with the mission of finding potential therapies that can be tested clinically in the near future.  We are an internationally supported group working in partnership with many similar organizations around the world.

It is our desire to advance research, education, improve the quality of life for the children affected by this disorder and ultimately find a cure.

EB Research Partnership

EB Research Partnership (EBRP) is working to treat and cure EB as quickly and efficiently as possible. We fulfill our mission by partnering with non-profit and for-profit entities and individual donors, as well as with the EB and research communities.

We are the largest nonprofit dedicated to funding research aimed at treating and ultimately curing EB, a group of devastating and life-threatening genetic skin disorders that affect children from birth.

Batten Disease Support and Research Association

Batten Disease Support and Research Association (BDSRA) is dedicated to funding research for treatments and cures, providing family support services, advancing education, raising awareness, and advocating for legislative action. Founded in 1987, by parents seeking to build a network for those diagnosed with Batten disease, BDSRA is now the largest support and research organization dedicated to Batten disease in North America.

BDSRA believes that to effectively unravel the mysteries of Batten disease, the worlds of medical science, research, and families must work together toward a common goal: discover treatments and cures while assuring a better quality of life for those living with the disease.

Garrett The Grand Batten Fighter

Garrett The Grand Batten Fighter (EBRP) September 5th 2014, the worst news ever given was learning Garrett’s diagnosis. Garrett has Neuronal Ceroid Lipofuscinosis, NCL, often called Batten Disease. His disease form is Infantile, INCL/CLN1.

This disease effects only 30 – 50 individuals a year in the United States and 2000 world wide. Within the next days, months and years Garrett will regress and will lose his speech, movement, cognitive abilities and have his life taken too early. He is currently losing vision, is almost blind and has many developmental delays. This disease will steal Garrett’s body and mind but for us lucky enough to know Garrett we will always know who he is.

Our goal is to educate and to have these rare diseases recognized. We need to get appropriate funding to end this unneeded pain for families.


Sanfilippo Children's Foundation

The Sanfilippo Children’s Foundation is dedicated to progressing clinical research into the effective treatment of Mucopolysaccharidosis III, also known as MPS III or Sanfilippo Syndrome.

Sanfilippo is a serious condition, delivering the most devastating outcome imaginable to patients of the disease and their families. But there is hope. There has been significant clinical progress in recent years and we believe that an effective treatment is within our reach. Much of this progress has been due to the tireless efforts of foundations and biotech companies established by families and researchers with the specific objective to pioneer treatments, taking them from “bench to bed” within accelerated time frames.


Sanfilippo Children's Research Foundation

The Sanfilippo Children’s Research Foundation – Entitled “A Life For Elisa” the ultimate goal of the foundation is to raise money to fund research and hopefully find a cure in Elisa’s life time – it is a daily crusade that the Linton family willingly charts.

Siblings Jessica and Connor, along with their parents, have devoted their lives to helping Elisa and bringing hope to every family who has a child afflicted with Sanfilippo syndrome. Their community of friends, medical specialists and local businesses have all joined in, surrounding this family with love, prayers, financial support, and volunteering in special ways, helping to raise over $5 million for research since 1999 with a remarkable 96% of every dollar donated being committed to research.

While Sanfilippo occurs once in 24,000 births, successful research into the disease could apply directly to related genetic conditions that affects 1 child in 5,000.


SAOIRSE foundation

The SAOIRSE foundation is a registered non-profit organisation established by Tony and Mary Heffernan in 2010. Named after their daughter, Saoirse, the charity originally focused on helping children suffering from rare genetic diseases, such as Batten Disease. We are active in advocacy and promotion of rare & genetic disorders and are actively involved in a number of national and international medical research and steering groups.

As the charity grew, it began to help more children in need. Now, through our 3 initiatives, we help critically ill children nationwide. If a child suffers from cancer, a chronic illness or a life limiting condition the Saoirse Foundation will offer as much support as we can. BUMBLEance provides much needed transport for these children, Liam’sLodge will offer respite for their entire family, and Bee for Battens supports all those affected by Batten Disease.

The Saoirse Foundation makes positive life impacts for sick children, and we will continue to help more critically ill children in the future.

Bee For Battens

Bee for Battens : Battens Disease is the Saoirse Foundation’s original initiative. It provides information and support to children and families affected by Battens Disease. We fund and aid the research of Battens Disease through fundraising and campaigns.

We are working to advance the education of the medical profession about Battens Disease and to promote global research into the management of this fatal neurological condition. Bee for Battens aims to raise awareness and fight Battens Disease worldwide.


Red Sanfilippo Fundacion

Red Sanfilippo Funacion (RS) – Only the power of the people can #curarsanfilippo. We are parents of children with a fatal disease that can be cured; we need help around the clock.

RS is established in 2012 by Janette Ojeda following the testimony of his son Iñaki with Sanfilippo syndrome, one in rare deadly disease for which there is currently no cure. Families, doctors and volunteers operated a non profit organization that aims to save the lives of children affected and adds to the work of other parents in different parts of the world and researchers working to develop treatment for the disease. We trust that arrive in time for our children and other children affected in Mexico and the world, saving their lives.


Fundacion Sanfilippo B

The B Sanfilippo Foundation defines its goals in the following objectives: 1. Disclose Sanfilippo disease to stop being a problem known by few and that it becomes a source of solidarity and knowledge by the Company 2. Encouraging research related to knowledge of the disease and possible treatment lines open. 3. To facilitate the dissemination of scientific advances that occur to be easily accessible to all interested persons 4. Participate with other associations or foundations projects of common interest.

Stop Sanfilippo Fundacion

The mission of Stop Sanfilippo is to encourage Sanfilippo Syndrome research with the objective of finding a cure or a treatment, improve the quality of life of the affected children and their families and spread knowledge about the illness in order to help achieve early diagnoses.


Fondation Sanfilippo

The purpose of the Foundation Sanfilippo Suisse is to help and develop ongoing research programs. All funding that the Foundation receives will be affected to promising new research programs, as well as the development and follow-up of ongoing research projects. In August 2008, we were informed that our little Charlotte, 4 years old, was affected by a rare, incurable and devastating disease, the Sanfilippo Syndrome. We soon discovered that she was far from being the only child afflicted with this genetic disease.