June 13, 2018 – Team Sanfilippo Foundation is proud to announce our collaboration with Lysosomal & Rare Disorders Research & Treatment Center (LDRTC) in Fairfax Virginia for a 6 month study of a potential drug candidate for Sanfilippo Syndrome. This will include testing for effectiveness across all types (A,B,C,D)
Testing will begin in July to screen a drug candidate in patient derived blood samples with Sanfilippo Syndrome (MPS-III A, B, C and D).”
The drug is being studied to prove its ability to clear undigested proteins from the lysosome by activating the Autophagy pathway (the body’s own garbage dump system).
We will be able to move quickly to patient studies if the research proves the clearance of proteins. We are hopeful for positive results that may lead to a viable treatment to lessen the burden of the disease.
We thank all our supporters for their ongoing fundraising efforts that allow us to fund research into clinically viable drugs to help our children in the short term. More help will be needed to fund a potential trial if we are successful and we will be ramping up fundraising to help us achieve our goals. Please contact us if you’d like to do fundraising for our Team. We cannot help our kids without your help.
This is THE FIRST time ALL TYPES of Sanfilippo will be participating in this type of study!!! This is so exciting for our community. If this proves successful, this is also not an age or regression restricted type of treatment. This is what we ALL could use as A TEAM!!! If you would like to speak to anyone about this, you can contact Kathy Buckley (President) at 518-879-6571, Patty Taormino (VP) 410-227-5711, and Danielle O’Connor (Treasurer and fundraising) 330-354-6401
We are SUPER EXCITED about this!!! Please share this announcement with everyone!! The more who see it, the more donations we get, the more children!
BioMarin Sanfilippo Syndrome Type B (MPS IIIB) Clinical Development Program
An Update for Patient Associations and Families
We would like to update you about the status of our clinical development program in Sanfilippo B. The BioMarin program, which involves multiple centers around the world, consists of three studies, described below.
The first study is an observational study of children with Sanfilippo B and includes testing of cognitive and adaptive function. The observational study lasts for 48 weeks. The tests explore how the child thinks and acquires new information as well as how the child deals with daily living. There are also assessments of behavior and quality of life. This study is intended to provide baseline information about how children with Sanfilippo B progress in the absence of treatment. This baseline information may then be compared to disease progression information from BioMarin’s treatment study (both for individual children and in aggregate).
The observational study is being conducted at sites in Australia, Colombia, Germany, Spain, Taiwan, Turkey, UK and USA. Enrollment to this study is now complete.
For more details, please visit: https://clinicaltrials.gov/show/NCT02493998
The second study is a treatment study in which children with Sanfilippo B will receive an investigational enzyme replacement therapy, known as BMN 250. The enzyme is administered directly to the brain as an infusion via a surgically implanted port. To enroll in the treatment study, a child must have completed the observational study as outlined above. This study is being conducted at the same centers. Children who have previously received an investigational drug as part of another clinical study, including gene therapy, may not be enrolled in this study.
For more details, please visit: https://clinicaltrials.gov/show/NCT02754076
The third study is a natural history study of children with Sanfilippo B up to 18 years old, who do not meet the criteria for participation in the observational study. Children enrolled into this study do not receive the investigational treatment. The objective of this study is to better understand the natural course of Sanfilippo B.
For more details, please visit: https://clinicaltrials.gov/ct2/show/NCT03227042
Your child’s doctor remains the best source of information regarding the care of your child and any questions or concerns should be directed to your child’s doctor.
If you represent a patient association, please contact BioMarin Patient Advocacy firstname.lastname@example.org. Alternatively, please email BioMarin Medical Information email@example.com
Updated: Dec. 2017 Below – We and several other foundations have funded and advanced more gene therapy trials with Abeona, and we at currently looking to finalize our contract and funding for this trial. Funds are critical to further these trial. A trial on average to treat 15-20 children costs average of 10 million. So again please.. these are trials, we need these to get done in order to get to hopeful FDA approved treatments. The cost is not cheap as we are so rare!! Drug companies don’t like investing in small populations. That’s why we need you!! Thank you!
UPDATE DEC. 2017
Enzyme replacement therapy
Hematopoietic stem cell transplantation
Small molecule(substrate reduction therapy, chaperones)
Team Sanfilippo grants $4,000,000 to open Phase 1B for three classes of patients; Children older than 7, children with AAV9 antibodies, and children previously treated with ert.
“Abeona is pleased to continue our global collaboration with the Sanfilippo foundations to help further advance our gene therapy programs for MPS III disease,” said Timothy J. Miller, Ph.D., president and chief executive officer of Abeona Therapeutics. “The effort and expertise that we continue to commit to the ABO-102 and ABO-101 programs puts us in a strong position to further extend the important progress reported to date. We are grateful to the foundations for their ongoing commitment to identifying and facilitating the development of clinical innovation to treat patients with MPS III disease.”
May 24th 2017 – ArmaGen breaks thru the BBB in humans with ERT! We’ve been watching this companies pipeline for 18 months now just waiting for the time to bring this treatment to Sanfilippo. While 2 other companies are dropping ERT trials, ArmaGen is trending in the most advanced process so far with minimal risk and side effect. Here is the latest report on what’s happening in another MPS disease that has been being treated for over 52 weeks now. http://armagen.com/news/trial-shows-armagen-biologic-improves-cognition-hurler-syndrome-patients/
This process looks promising for so many neurocognitive diseases. Please read our latetest in “Whats Happening” on how you can help. We have partnered with Armagen and have met with FDA to move this forward to our Sanfilippo kids by years end or first quarter of 2018!
Team Sanfilippo is preparing to do a 6 month research study on MMJ for sleep, dystonia and hyperactive behavior. We would like to see if there is any interest in our Sanfilippo community. The criteria is that you have to live in MMJ legal state, not be on MMJ currently, have a MMJ doctor to prescribe and follow patient and have one of the three symptoms. We feel that if we could have a published research specifically on improvements with Sanfilippo then we could get better access to MMJ.
Please post if you are interested and send Kathleen an email firstname.lastname@example.org
We constantly are working on improving quality of life and we believe that MMJ may be able to improve sleep and movement disorders and we hope to be able to prove it through a clinical study.
Ten Sanfilippo Families Needed for Pilot Testing of Genetic Screening Program
We at Team Sanfilippo have partnered with DNAcheckup, a new nonprofit organization, whose goal is to provide genetic data through saliva testing to families of patients diagnosed with recessive genetic disorders. This pilot program is for a limited amount of families and the initial requirement for the ten families is that the patient must have already obtained a genetic screening, so their mutations are known. From the patient’s data, family members (mother, father, siblings, uncles, aunts and cousins) will be tested for just the mutations known in the patient. This data can be used to determine (in coordination with your physician or genetic counselor) the family member’s carrier status for family planning decisions, like the utilization of in vitro fertilization. The pilot program is meant to give DNAcheckup a test run of their processes and to provide potential funders with testimonials and assurance that their services are needed and are working properly. This pilot program will be able to help DNAcheckup refine their processes and feedback from participants is essential. In 2017, DNAcheckup plans to raise funds to provide free testing to many more families of patients with recessive genetic disorders. The costs of the testing (saliva kit, shipping and lab testing) will be free to the pilot program participants. The pilot program is open to US families but in the future DNAcheckup will provide testing globally. To find out more about participating, please contact email@example.com directly and they will call you with additional details and logistics. We are excited to fund the pilot program testing costs for our patient families in partnership with DNAcheckup and look forward to when DNAcheckup can expand this program to ALL of our families. For further information about the pilot program and DNAcheckup go to http://www.dnacheckup.org/wp-content/uploads/2016/08/DNAcheckup.pdf
We are so happy this is finally happening after the research that started out 18 years from one family organization, “Ben’s Dream”. Unfortunately their son never got see the treatment. In Honor of Benjamin Siedman, we pray this therapy works! For the past 10 years Team Sanfilippo and many others have organized to pool our money together for this therapy! Now it is time to continue on for treatments for quality of life for ALL the kids who do not qualify for this trial or treatment! This one thing Team Sanfilippo has always and will always do, is work for treatments for ALL Sanfilippo kids at different stages of the disease, not just a cure the young one! The family foundations who have worked so hard over the many years can be happy for future generations, but as for their own children they will never see the cure and those who are still with us, still continue to decline and all desperately need quality of life treatments. Please continue to fund raise, donate and share with others, as watching the suffering of these children while the doctors having no answer is cruel. We at TSF work to fill that gap!
Biomarin enrolls first patient with Sanfilippo B ….
Abeona Therapeutics Announces Initial European Regulatory Approvals for Phase 1/2 Gene Therapy Clinical Studies for Patients With Sanfilippo Syndromes Type A (MPS IIIA) and Type B (MPS IIIB)
Abeona Therapeutics January 11, 2016 9:03 AM
- Approval of Genetically Modified Organism (GMO) and Ethical Committee (CEIC) regulatory filings for both ABO-101 (AAV-NAGLU) and ABO-102 (AAV-SGSH) in Spain
- Phase 1/2 dose escalation studies of ABO-101 and ABO-102 in Sanfilippo syndrome types A and B
- Natural history study in 25 patients established efficacy endpoints
Abeona Therapeutics, Inc. (ABEO), a biopharmaceutical company focused on developing and delivering gene therapy and plasma-based products for severe and life-threatening rare diseases, today announced the Interministerial Council of Genetically Modified Organisms has approved the Genetically Modified Organism (GMO) Voluntary Release regulatory filings for both Phase 1/2 Gene Therapy Clinical Studies to treat patients with AB0-101 (AAV NAGLU) and ABO-102 (AAV SGSH) for patients with Sanfilippo syndrome type A (MPS IIIA) or type B (MPS IIIB). Additionally, the Comite Etico De Investigacion Clinica de Euskadi (CEIC-E) has approved the ethical committee regulatory filings for both ABO-101 and ABO-102. The Company plans to file CTAs for both programs shortly for the upcoming clinical studies to be conducted at Cruces University Hospital (Bilbao, Spain).
“The GMO and Ethical committee approvals — which are similar to the Recombinant DNA Advisory Committee (RAC) and hospital Institutional Review Board processes in the United States — support our planned path to clinical trials in the EU for the treatment of Sanfilippo syndromes type A and B. Pre-clinical studies have demonstrated that a single intravenous injection of ABO-101 or ABO-102 restored the enzyme activity in the cerebral spinal fluid and serum, and also corrected the lysosomal storage pathology throughout the CNS and body,” noted Tim Miller, PhD, President & CEO. “We look forward to advancing these potentially life-changing therapies into global clinical trials and grateful for the support and guidance from regulators, clinicians, and patient community.”
“Sanfilippo syndromes type A and B are devastating lysosomal storage diseases that affect children around the world. These gene therapies, delivered as a single, non-invasive intravenous injection, offer a new and promising treatment paradigm for patients with this relentless disease,” commented Luis Aldámiz-Echevarría, M.D., PhD., Principal Investigator of the clinical trials and Associate Professor in the Faculty of Medicine at the University of the Basque Country (Spain), Paediatrician in the Department of Paediatrics at Cruces University Hospital (Spain) and Principal Investigator of the Metabolic Inherited Disorders Group and the Metabolomics and Proteomics Platform at BioCruces Health Research Institute (Spain).
Sanfilippo syndromes (or mucopolysaccharidosis (MPS) type III) are a group of four inherited genetic diseases each caused by a single gene defect, described as type A, B, C or D, which cause enzyme deficiencies that result in the abnormal accumulation of glycosaminoglycans (sugars) in body tissues. MPS III is a lysosomal storage disease, a group of rare inborn errors of metabolism resulting from deficiency in normal lysosomal function. The incidence of MPS III (all four types combined) is estimated to be 1 in 70,000 births. Mucopolysaccharides are long chains of sugar molecule used in the building of connective tissues in the body. There is a continuous process in the body of replacing used materials and breaking them down for disposal. Children with MPS III are missing an enzyme which is essential in breaking down the used mucopolysaccharides called heparan sulfate. The partially broken down mucopolysaccharides remain stored in cells in the body causing progressive damage. Babies may show little sign of the disease, but as more and more cells become damaged, symptoms start to appear. In MPS III, the predominant symptoms occur due to accumulation within the central nervous system (CNS), including the brain and spinal cord, resulting in cognitive decline, motor dysfunction, and eventual death. Importantly, there is no cure for MPS III and treatments are largely supportive care.
About ABO-101 (AAV NAGLU) and ABO-102 (AAV SGSH) are next generation adeno-associated viral (AAV)-based gene therapies for MPS IIIA and MPS IIIB, respectively. These gene therapies involve a one-time delivery of a genetically modified virus to deliver a normal copy of the defective gene to cells of the central nervous system and peripheral organs with the aim of reversing the effects of the genetic errors that cause the disease. After a single dose in preclinical animal models of Sanfilippo syndrome, ABO-101 and ABO-102 induced cells in the CNS and peripheral organs to produce the missing enzymes and help repair damage caused to the cells. Preclinical in-vivo efficacy studies in animals with Sanfilippo syndrome have demonstrated functional benefits that remain for months after treatment. A single dose significantly restored normal cell and organ function, corrected cognitive defects that remained months after drug administration, increased neuromuscular control and increased the lifespan of animals with MPS III over 100% one year after treatment compared to untreated control animals. These results are consistent with studies from several laboratories suggesting AAV treatment could potentially benefit patients with for Sanfilippo syndrome Type A and B, respectively. In addition, safety studies conducted in animal models of Sanfilippo syndromes have demonstrated that delivery of AB0-101 and ABO-102 are well tolerated with minimal side effects.
About Abeona: Abeona Therapeutics, Inc. develops and delivers gene therapy and plasma-based products for severe and life-threatening rare diseases. Abeona’s lead programs are AB0-101 (AAV NAGLU) and ABO-102 (AAV SGSH), adeno-associated virus (AAV)-based gene therapies for Sanfilippo syndrome (MPS IIIB and IIIA). We are also developing ABO-201 (AAV CLN3) gene therapy for juvenile Batten disease (JBD); and ABO-301 (AAV FANCC) for Fanconi anemia (FA) disorder using a novel CRISPR/Cas9-based gene editing approach to gene therapy program for rare blood diseases. In addition, we are also developing rare plasma protein therapies including SDF Alpha™ (alpha-1 protease inhibitor) for inherited COPD using our proprietary SDF™ (Salt Diafiltration) ethanol-free process. For more information, visit www.abeonatherapeutics.com.
This press release contains certain statements that are forward-looking within the meaning of Section 27a of the Securities Act of 1933, as amended, and that involve risks and uncertainties. These statements include, without limitation, our plans for continued development and internationalization of our clinical programs, management plans for the Company, and general business outlook. These statements are subject to numerous risks and uncertainties, including but not limited to continued interest in our rare disease portfolio, our ability to enroll patients in clinical trials, the impact of competition; the ability to develop our products and technologies; the ability to achieve or obtain necessary regulatory approvals; the impact of changes in the financial markets and global economic conditions; and other risks as may be detailed from time to time in the Company’s Annual Reports on Form 10-K and other reports filed by the Company with the Securities and Exchange Commission. The Company undertakes no obligations to make any revisions to the forward-looking statements contained in this release or to update them to reflect events or circumstances occurring after the date of this release, whether as a result of new information, future developments or otherwise.
- Health Care Industry
- Sanfilippo syndrome
- Genetically Modified Organism
- gene therapy
- Clinical Studies
Company and Media Andre’a Lucca Vice President of Communications Abeona Therapeutics, Inc. +1 (212)-786-6208 firstname.lastname@example.org Magdalena Tyrpien Director of Investor Relations Abeona Therapeutics, Inc. +1 (212)-786-6209 email@example.com
The Company is well positioned to execute on its strategy to become a leader in cell therapy and gene therapy for rare diseases
- Abeona’s preclinical studies have demonstrated promising efficacy in cognitive function, motor, and survival in Sanfilippo Syndrome (MPS IIIA and MPS IIIB)
- Two Phase 1/2 clinical trials for ABX-101 (MPS IIIB) and ABX-102 (MPS IIIA) for Sanfilippo Syndrome to commence in 2015
PlasmaTech Biopharmaceuticals, Inc. (PTBI), a biopharmaceutical company advancing protein biologic therapies and oncology supportive care products, announced today that it has entered into a definitive agreement to acquire Abeona Therapeutics, a company engaged in the development and commercialization of therapies for patients with lysosomal storage diseases. Under the terms of the agreement, the Company will issue to Abeona Therapeutic members a total of 3,979,761 common You can read more here http://finance.yahoo.com/news/plasmatech-biopharmaceuticals-announces-agreement-acquire-100300088.html
Watch Tim Miller’s interview here https://vimeo.com/126837840
FOR IMMEDIATE RELEASE Abeona Therapeutics Raises Foundation-led $3.6M
Tim Miller, PhD President & CEO
Abeona Therapeutics -216-445-4668
FOR IMMEDIATE RELEASE Abeona Therapeutics Raises Foundation-led $3.6M to Develop Therapies for Children Suffering from Rare Sanfilippo Diseases Cleveland, OH, December 1, 2014 – Biotechnology startup Abeona Therapeuticsannounced today it closed $3.6 million to complete preclinical development of therapies for children with Sanfilippo syndrome (mucopolysaccharidosis type III, MPS III), a rare terminal genetic disorder that results in death in children before they reach their mid-teens. Investors include U.S.-based Cure Sanfilippo Foundation, Sanfilippo Research Foundation (SRF), Team Sanfilippo, the Abby Grace Foundation, and the National MPS Society. Abeona also generated strong support from the international Sanfilippo community, with investments from Spain-based Stop Sanfilippo and Sanfilippo B Foundation, Geneva, Switzerland-based Fondation Sanfilippo and Mexico-based Red Sanfilippo Foundation. Support for the program has also been provided by The Children’s Medical Research Foundation, Inc. (CMRF) and Canadian led Sanfilippo Children’s Research Foundation (SCRF).
“For over a decade, our Sanfilippo research has been supported by Ben’s Dream – SRF, and Cure Kirby – CMRF, and jointly by A Life for Elisa – SCRF, Team Sanfilippo and LivLife Foundation since 2010. We are very thankful that our joint efforts have led to our planned clinical trials in 2015,” said Drs. Douglas McCarty and Haiyan Fu, Abeona’s scientific founders and investigators at Nationwide Children’s Hospital.”
Sanfilippo syndrome is a group of 4 deadly genetic diseases resulting from the body’s inability to properly break down certain sugars. Symptoms often appear in the first year of life, and the disease causes progressive muscular and cognitive decline in children after the age of two. There is no cure and currently no approved treatments for Sanfilippo syndrome. As such, children afflicted with Sanfilippo syndrome experience progressive loss of speech, the ability to eat and walk, and rarely live past their second decade of life. Abeona Therapeutics is focusing initially on gene therapies for Sanfilippo syndrome types A and B.
“This funding helps support our global development of gene therapies for treating patients with Sanfilippo syndrome. We have formed a very special, international, collaborative effort where Abeona has become the voice of over a dozen patient advocacy groups desiring accelerated access to potential therapies,” explained Tim Miller, Ph.D, Abeona’s President/CEO. “We are pleased to collaborate with Nationwide Children’s Hospital in the development of these therapies as we approach clinical trials.”
“We are excited to be working with Abeona and the impressive world-wide collaboration of Sanfilippo foundations they have organized. Their emphasis on developing minimally-invasive treatments is reassuring as a parent. The dedication and approachability of the team has built confidence in the program and allows foundations to feel connected to the pursuit of a cure,” said Kathleen Buckley, President of the Team Sanfilippo Foundation.
Time is a critical factor for children with Sanfilippo syndrome: 70% of the children with the disease will not reach their 18th birthday. “We are poised to initiate clinical trials in early -to-mid 2015 for these much needed therapies,” said Kevin Flanigan, MD, principal investigator, Center for Gene Therapy, and neurologist at Nationwide Children’s Hospital and Professor of Pediatrics and Neurology at The Ohio State University College of Medicine. He is also principal investigator for the ongoing Sanfilippo Natural History Study and upcoming gene transfer trials at Nationwide Children’s.
The collaborative efforts of Abeona, the supporting Sanfilippo foundations, and Nationwide Children’s Hospital were recognized by receipt of the Global Genes “RARE Champion of Hope” Collaborations in Advocacy award in late 2013 and by recent support from the National MPS Society. “The mission of the National MPS Society is to support promising therapies for these devastating, rare diseases, and we are pleased to support Abeona’s progress towards clinical trials, ” said Barbara Wedehase, Executive Director.
Cleveland-based Abeona Therapeutics is a start-up company focused on developing gene therapy based treatments for patients with lysosomal storage diseases, with lead products focusing on MPS IIIA and IIIB. Abeona Therapeutics received Orphan Product Designations for MPS IIIA and IIIB in 2014 and clinical trials are anticipated to begin in early-to-mid 2015.
About Abeona Therapeutics 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. 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. Clinical trials for Sanfilippo types A and B are anticipated to begin in early-to-mid 2015.
About Nationwide Children’s Hospital Ranked 7th of only 10 children’s hospitals on U.S. News & World Report’s 2014-15 “America’s Best Children’s Hospitals Honor Roll” and among the Top 10 on Parents magazine’s 2013 “Best Children’s Hospitals” list, Nationwide Children’s Hospital is one of the nation’s largest not-for-profit freestanding pediatric healthcare networks providing care for infants, children and adolescents as well as adult patients with congenital disease. As home to the Department of Pediatrics of The Ohio State University College of Medicine, Nationwide Children’s faculty train the next generation of pediatricians, scientists and pediatric specialists. The Research Institute at Nationwide Children’s Hospital is one of the Top 10 National Institutes of Health-funded free-standing pediatric research facilities in the U.S., supporting basic, clinical, translational and health services research at Nationwide Children’s. The Research Institute encompasses three research facilities totaling 525,000 square feet dedicated to research. More information is available at NationwideChildrens.org/Research.