May 12, 2014 -Abeona Therapeutics Receives U.S. Orphan Drug Designations for treatment of Sanfilippo Syndromes A and B

Team Sanfilippo along with numbers of other patient advocate groups and organizations are continuing to raise much needed funds to advance its gene therapy-based clinical programs for both Sanfilippo Syndrome type A and B. Phase I/II clinical trials for both diseases are anticipated to begin in 2014 now we have approval.

Abeona Therapeutics – named after the Roman Goddess who is the protector of children – was formed in early 2013 to help focus the search for a cure for Sanfilippo Syndrome and provide a unifying voice between patient advocate groups, researchers, clinicians and investors. Abeona Therapeutics is the result of collaborative efforts between Nationwide Children’s Hospital and multiple international patient advocate groups for developing Sanfilippo therapies, including The Children’s Medical Research Foundation, Inc. (USA), Team Sanfilippo (USA), Fondation Sanfilippo (Switzerland), Stop Sanfilippo (Spain), Ben’s Dream: The Sanfilippo Research Foundation (USA), and the Sanfilippo Children’s Research Foundation (Canada). The collaboration has helped focus parents and caregivers on a leading therapy with broad potential to provide long-term benefits to children with Sanfilippo Syndrome.

read more here…. http://news.yahoo.com/abeona-therapeutics-receives-u-orphan-drug-designations-treatment-130000138.html

Just this week, the FDA grants Abeona Therapeutics’ two gene therapies for Sanfilippo Syndrome Types A and B, orphan drug designation. No gene therapy has ever been approved for sale in the United States. In Europe, the EMA (European Medicines Agency), has given regulatory approval to UniQure, to sell its gene therapy, Glybera, for the treatment of the rare disease, Lipoprotein Lipase Deficiency (LPLD. To read momre ……. http://orphandruganaut.wordpress.com/2014/05/01/sanfilippo-syndrome-fda-orphan-designations-for-gene-therapy/ Thank you for the continued support donations to bring the trial forward for the children and families!!

On Wednesday March 12, 2104 in Bethesda, MD. We joined the researchers & doctors from Nationwide Children’s Hospital, Abeona Therapeutics, and other Sanfilippo Parent founded organizations and families for a key step in moving the program forward. The RAC submission to the NIH was approved by a vote of 17 -0. This means the program moves one step closer to FDA approval for a clinical trial for Type B. This also paves the way for type A to move forward close behind. It was wonderful that so many families were able to meet together with all those on the research end as well. The public comments given by the Wilson’s, the Linton’s, the Seidman’s and Cara O’Neill were all very moving. Thank you to all the families that attended in support!

The fundraising must continue as we move forward with the trials. So please help us to finish this project, as it the most promising for the children! We need this treatment now, so let’s march forward and finish it!!

Quartet of advocacy groups seed an Ohio startup’s gene therapy for rare Sanfilippo syndrome

December 16, 2013

A Cleveland biotech startup focused on a rare genetic disease provides an interesting example of the growth of alternative funding for life science companies.

Abeona Therapeutics’ just-announced $750,000 seed round, which will allow the startup to complete preclinical development of gene therapies for Sanfilippo syndrome, came not from angel or venture capital investors but from research and advocacy groups. This is the start up needed to get trials going, but still need this amount or more to continue thru the end of trials.

Natural History Study In Mucopolysaccharidosis Type III

Mucopolysaccharidosis (MPS) type III, also known as Sanfilippo syndrome, is a group of four genetic diseases. We are conducting a natural history study in MPS III. We will enroll a total of 30 subjects in this study; 15 subjects with MPSIIIA and 15 subjects with MPSIIIB.

Principal Investigator:
Kevin Flanigan, MD

Co-investigators:
Kim McBride, MD
Haiyan Fu, PhD
Doug McCarty, PhD
Keith Yeates, PhD
Marco Corridore, MD

Mucopolysaccharidosis (MPS) type III, also known as Sanfilippo syndrome, is a group of four genetic diseases. These diseases result in a buildup of specific sugars in the brain and spinal cord. These diseases cause a decrease in mental function and the ability to move. They eventually result in death.

We are looking for patients for a study of the natural history of MPS type III, with the following goals:

To find how fast mental function and the ability to move decreases
To study the natural history and progress of the disease
To establish normal ranges of function in patients who will be potential subjects for a future treatment study using gene therapy
To find indicators of disease progression over one year. This will include changes in brain activity and in cerebrospinal fluid

Study Population

We will enroll 30 subjects total in this study. We will enroll 15 subjects with MPSIIIA and 15 subjects with MPSIIIB. All subjects must meet the following criteria:

Must be at least 2 years old
Must have genetic testing that confirms a diagnosis of MPSIIIA or MPSIIIB
Must have symptoms of the disease including mental decline
Must be able to participate in three clinical visits over the course of 1 year
Must not be taking any medication that prevents a spinal tap or use of anesthetics
Must be able to be safely sedated in the opinion of the clinical anesthesiologist

For more information regarding this study, please contact the Study Coordinator Krista Kunkler at 614-722-2238, or by email at Krista.Kunkler@nationwidechildrens.org.

This study will be an asset in moving forward with the Gene Therapy with Abeona ! Exciting times are here!!

Abeona Therapeutics Closes $750,000 in Seed Round to Develop

Therapies for Children Suffering from Rare Sanfilippo Diseases

It’s been an exciting few months for Abeona. We successfully closed our $750,000
seed round financing in December 2013 to help complete all pre-clinical studies
and regulatory submissions necessary to achieve active Investigational New Drug
(IND) applications with the FDA. We anticipate these studies will be completed
in early 2014 and anticipate enrolling patients in Phase I/II trials for both of
the IIIA and IIIB programs in 2014. Keeping on track with our development
timelines, the clinical team has already submitted the necessary paperwork with
the NIH Recombinant DNA Advisory Committee for review in preparation of our IND
submission. We kicked off our Series A fundraising campaign at the JP Morgan
conference, held annually every January in SanFrancisco, in order to raise funds
for both IIIA and IIIB clinical trials. Over the next few months, we look
forward to providing everyone with updates on the regulatory milestones, drug
manufacturing and clinical trial progress.

We (the parent organized foundations) will be needing to continue raising funds as the gene therapy trials move forward in patients, so please, this is not an end to the needed funds. These trials will need additional funding as we follow the patients during the trial. So any fund raisers or donations are still greatly needed!!

January 20, 2014 – Villanova Teaming Up to Raise Awareness for Sanfilippo Syndrome at Jan. 20 Men’s Basketball Game

@ Wells Fargo Center

Villanova, PA

Press Release > http://www1.villanova.edu/villanova/media/pressreleases/2014/0110-1.html

To purchase tickets > http://www.villanova.com/tickets/m-baskbl-tickets.html

Villanova versus Creighton will be “Sanfilippo Syndrome Awareness Night,” with several planned activities aimed at raising awareness for the rare disorder