BBDF and Theranexus collaborate to publish journal

BBDF and Theranexus collaborate to publish “Evaluating and modulating TFEB in the control of autophagy: toward new treatments in CNS disorders” in the journal Fundamental & Clinical Pharmacology

Intense scrutiny of patient cells and animal models of CLN3 disease over the past 3 decades demonstrates that the loss of CLN3 protein impacts multiple cellular functions. The majority of dysfunction is in cellular compartments responsible for cellular recycling and waste management. This review, co-authored by Beyond Batten Disease Foundation and Theranexus, describes how drug-mediated activation of transcription factor EB (TFEB) clears toxic waste from Batten and other neurodegenerative disease models and describes ongoing pharmaceutical plans to use TFEB activators to treat these diseases.

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Manuscript accepted by the journal

NEUROPHARMA: Evaluating and modulating TFEB in the control of autophagy: towards new treatments in CNS disorders

Manuscript by da Costa, Anaelle; Metais, Thibaud; Mouthon, Franck; Kerkovich, Danielle; Charvériat, Mathieu, was successfully accepted by the journal “Fundamental & Clinical Pharmacology.”

BBDF-101 Announcement

We are excited to announce the following major milestone for BBDF!

The FDA has awarded Orphan Drug and Rare Pediatric Disease designations to Beyond Batten Disease Foundation for BBDF-101. The aim of the program is to facilitate the development of new drugs and biological products for the prevention and treatment of rare pediatric diseases. These designations provide accelerated review for approval, support with the FDA regulatory process and at least seven years of post-approval protection, as well as exemption from filing fees that normally have to be paid to the FDA. These designations do not impact the clinical trial, but are beneficial once the therapy receives New Drug Approval. We are grateful to Theranexus for their continued partnership and commitment to initiating a clinical trial for BBDF-101 as quickly as possible.

THERANEXUS AND BBDF OBTAIN ORPHAN DRUG DESIGNATION (ODD) AND RARE PEDIATRIC DISEASE DESIGNATION (RPDD) FROM THE FOOD AND DRUG ADMINISTRATION (FDA) FOR BBDF-101 FOR BATTEN DISEASE

  • Orphan Drug Designation (ODD) is a status that provides seven years of additional post-approval protection and exemption from filing fees
  • Rare Pediatric Disease Designation (RPDD) qualifies the sponsor at the time of registration for a salable, transferable priority review voucher which can be used to speed up the approval process for another drug candidate

Lyon, August 28, 2020 – Theranexus, a biopharmaceutical company innovating in the treatment of neurological diseases, and Beyond Batten Disease Foundation (BBDF) today announced the decision by the Food and Drug Administration (FDA) to award Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation (RPDD) to the drug candidate BBDF-101 for Batten disease, a rare, fatal, genetic disorder of the nervous system for which there is no treatment.

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BBDF-101 Announcement

We are excited to announce the following major milestone for BBDF! Theranexus, a publicly-traded European pharmaceutical company, has committed the $20 million and expertise necessary to complete the clinical trial and commercialization for our drug discovery, BBDF-101.

Theranexus and Beyond Batten Disease Foundation (BBDF) announce the signing of a worldwide exclusive license for Batten disease drug candidate BBDF-101
  • The agreement covers the development and marketing of the drug following a single clinical trial due to begin in 2020. 
  • If successful, this trial will lead directly to the drug’s approval.

Lyon, 12 December 2019 – Theranexus, a biopharmaceutical company innovating in the treatment of neurological diseases and pioneer in the development of drug candidates modulating the interaction between neurons and glial cells, is pleased to announce the signing of an agreement with Beyond Batten Disease Foundation, granting it a worldwide exclusive license to develop and commercialize the drug candidate BBDF-101 for juvenile Batten disease.

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A Mother’s Story

 

Photo by Catherine Sanderson | catherinesanderson.com
On March 28, 2008, my husband Craig and I received the devastating news that our daughter, Christiane had been diagnosed with a very rare neurodegenerative disease called Batten Disease. Batten Disease causes blindness, seizures, is physically and mentally incapacitating and is ultimately terminal by the late teens or early 20’s. Seven years later, it still takes my breath away to write all of that in one sentence.

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2nd Annual Bind Up Batten Golf Charity

Bind Up Batten brought together the community on July 20, 2019, for the 2nd Annual Batten Disease Golf Charity Outing. Over 80 golfers came out on a beautiful, cloudy day in Waterville, Ohio to raise $12,700 for juvenile Batten Disease research.

In addition to the golfers, Bind Up Batten would like to thank the families, friends, musicians, Fallen Timbers Fairways and all the sponsors who provided their support to help make this event happen.

Special thanks and recognition to Mike’s parents TJ and John Klein as well as Donna and Paul Iwinski for their incredible effort along with many other volunteers who made this day possible.

Together we can Bind Up Batten!

Watch the video

We are Austin Interview with Garland Benson

We Are Austin did an interview highlighting Garland Benson and the Be a Hero Campaign

Check out his interview here:

If you had a goal to raise one million dollars in two years, you might think it would be impossible! Now imagine you’re 12 years old and setting out on this incredible mission. Well, Garland Benson did just that, and his perseverance will help fund research to treat his older sister’s degenerative disease. Garland is here this morning to talk about his remarkable success raising funds for Beyond Batten.

Thanks again to everyone who helped Garland hit his goal!

BBDF is actively pursuing a pharmaceutical partner to support the clinical trial and commercialize the treatment.  Donations are rolling in and every additional dollar raised by Garland’s campaign will help offset activities required by the FDA to start the trial.

Donate today.

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