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.
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.
Kevin Flanigan, 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
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
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
11471 Euclid Ave, Ste 211
Cleveland OH 44106
Abeona Therapeutics, Inc. is developing gene therapy based potential cures for the deadly childhood diseases Sanfilippo (SF) Syndrome Type A and Type B. In SF disease, the predominant symptoms occur due to improper cell function within the central nervous system (CNS), which result in cognitive decline, motor dysfunction, and eventual death. Our two lead products, ABX-A and ABX-B, uniquely deliver the therapeutic product to the CNS with the aim of reversing the effects of the genetic errors that cause the disease. ABX-A and ABX-B induce cells in the CNS and digestive tract to produce the missing enzymes and help repair damage caused to the cells.
Safety studies conducted in large animal models have demonstrated that delivery of ABX-A and ABX-B are well tolerated with minimal side effects. Importantly, efficacy studies in animals with Sanfilippo syndrome have demonstrated unprecedented therapeutic benefit months after treatment. A single dose of ABX-A or ABX-B significantly restored normal cell and organ function and increased the lifespan of animals with SF over 100% a year after treatment compared to untreated control animals. Similarly, animals treated with ABX-A and ABX-B demonstrated significant corrections of cognitive defects that remained months after drug administration. These results are consistent with studies from several laboratories suggesting ABX-A and ABX-B treatment could potentially benefit patients with for Sanfilippo Syndrome Type A and B, respectively.
Abeona, in partnership with Nationwide Children’s Hospital (Columbus, OH), has met with the FDA and is planning to initiate clinical studies of ABX-A and ABX-B in patients with Sanfilippo Syndrome Type A and B set to begin in 2014.
LYSOGENE ANNOUNCES COMPLETE ENROLLMENT IN ITS SAF-301 PHASE I/II CLINICAL TRIAL OF INTRACEREBRAL GENE
THERAPY IN CHILDREN WITH SSANFILIPPO TYPE A SYNDROME.
Paris, France – June 14, 2012 – LYSOGENE announced today that the last planned patient in its phase
I/II clinical trial in Sanfilippo Type A Syndrome (NCT01474343) had been treated with SAF-301, its
investigational intracerebral gene therapy product.
SAF-301 aims at treating this pediatric life threatening disease with a high unmet medical need and
currently no cure.
This open-label, single arm, monocentric, phase I/II SAF-301 clinical study is primarily designed to
evaluate the tolerance and the safety of the intracerebral administration of SAF-301 performed in a
single neurosurgical session. It is also designed to evaluate exploratory efficacy neuropsychological,
radiological and biological endpoints from the perspective of future pivotal studies.
“Completing full enrollment and treatment in this phase I/II study is a crucial milestone in the
development of a safe and efficacious gene therapy as what is intended to become the first line
treatment for Sanfilippo Type A disease. It also sustains the extraordinary potential of gene deliverybased
approaches for numerous other monogenic diseases with central nervous system involvement.
Our hope is to bring significant clinical benefits and quality of live improvements to numerous
patients and their families affected with such diseases worldwide”, Karen Aiach, Founder and CEO of
About Sanfilippo Syndrome and LSDs
Sanfilippo Syndrome or Mucopolysaccharidosis III (MPS-III) is a group of four rare autosomal
recessive lysosomal storage diseases of which Type A accounts for approximately two thirds.
Sanfilippo Syndrome is characterized with a heavy central nervous system involvement and an
extremely severe phenotype, associated with a life expectancy reduced to the midst of the second
Sanfilippo Type IIIA Syndrome is seen in approximately 1 in 100,000 live births and affects a few
thousands patients worldwide.
MPS-III belongs to lysosomal storage diseases (LSDs), a group of over 50 inherited disorders, with a
total combined incidence greater than 1 per 8,000 births, of which 70% have a central nervous
LYSOGENE is a platform biotechnology company specialized in the development of intra-cerebral
gene therapy for the treatment of lysosomal diseases affecting the central nervous system, the main
cause of mortality in childhood neurodegenerative diseases.
LYSOGENE brought its first product SAF-301 from bench to the bedside in less than five years.
LYSOGENE – 52, rue la Boétie – 75008 Paris – France – www.lysogene.com