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: This study is not yet open for participant recruitment


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:


United States

National MPS Society Logo

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 Logo

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 Logo

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.

Cure Sanfilippo Foundation Logo

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.

Jared's Fight Logo

Jared’s Fight – Meet Jared Connor. He is seven years old. Jared has been diagnosed with a rare disease. He has Sanfilippo Syndrome. Children with Sanfilippo are missing an essential enzyme that breaks down a complex body sugar called heparin sulfate. These sugars slowly build up in the brain, stopping normal development and causing hyperactivity, sleep disorders, loss of speech, dementia and typically death before adulthood. Other complications include blindness, seizures, mental retardation and progressive neurological disease leading to becoming wheelchair bound and the inability to care for oneself.

LivLife Logo

LivLife is a not for profit foundation dedicated to the fight against MPS disorders through funding cutting edge research, educating the community and supporting families affected by MPS disorders. Inspiring others to LIV their best life. To influence optimism and love in any circumstance

National MPS Society Logo

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.

SF4K Logo

SF4K (Sanfilippo Foundation for Children) – We are single-mindedly devoted to finding a cure to Sanfilippo Syndrome through gene therapy. In layman’s terms, gene therapy uses a biologic agent (like a virus) to insert functional copies of the defective gene into affected patients. Once inserted, copies of the gene cause the body to continually express the missing enzyme. The enzyme produced by the newly inserted functional copies of the gene cause the body to clear the accumulated waste material. For very rare diseases like Sanfilippo (which affects roughly 1 in 70,000 births), it is up to patient advocates to drive the science. Big pharmaceutical companies are also typically not in the business of developing once in a lifetime cures.

Team Sanfilippo LogoTeam Sanfilippo Logo

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 (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.

Team Sanfilippo Logo

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.


Sanfilippo Children's Foundation Logo

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


Sanfilippo Children's Research Foundation Logo

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


Red Sanfilippo Logo

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.


Little Maciek Logo

Little Maciek and Great Wizards – Maciek is 6 years old. He loves sliding, sand games, the company of other children and hugging his parents. He is a really big eater and loves Tom & Jerry cartoons. He is also dying. Maciek was diagnosed a year ago with mucopolysaccharidose, type III A, known also as the Sanfilippo syndrome. This is a very rare genetic disease, occurring once in every 100 thousand births. It is caused by a metabolic disorder leading to storing the sugar molecules, called mucopolysaccharides, that are not properly disposed of from the body. As a result the body cells and organs are being damaged and eventually the whole body of the child is destroyed.


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.

National MPS Society Logo

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 Logo

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.