Beyond Batten Disease Foundation


   

TRAIN Inventory Profile

Mission
Strategy/Approach
Research Portfolio
Financials
Key Accomplishments
Leadership
Ask Us About
 EXPAND ALL

Mission

Beyond Batten Disease Foundation (BBDF) is the world’s largest organization dedicated to funding research for a treatment or cure for juvenile Batten disease. Batten disease is a rare, inherited pediatric neurological disorder, which begins with vision loss and seizures, followed by cognitive and motor impairment, and ultimately death by the late teens or 20s. Since its inception in 2008, BBDF has invested over $15.5 million for research through by leveraging donations, co-funding, and partnerships.

Strategy/Approach

BBDF is spearheading a unique, cohesive strategy, incorporating independent scientific resources and collaboration with related organizations to drive research in Batten Disease.

Research Portfolio

Today, BBDF is advancing the development of six potential drug compounds for treatment. To prepare for success, we are supporting the coordination and continued development of patient registries around the globe. Additionally, we are providing Batten-specific resources to individual investigators as well as the Pharmaceutical Industry to explore the potential of new discoveries to treat juvenile Batten disease. Our research strategy brings together all stakeholders, including affected families, like-minded foundations, and other neurodegenerative disease groups. BBDF Principal Scientist Danielle Kerkovich, PhD, together with experts in all levels of drug discovery and approval, continues to lead and manage our comprehensive research strategy, including over 38 concurrent projects, to ensure that we stay on track.

Financials

Fiscal year 2014, year ending 6/30/2015

  • Revenue: $1,388,871
  • Assets: $129,397
  • Grants: $22,000
  • Gifts Received: $1,366,871
  • Total Expenditures: $1,928,094 

Key Accomplishments

BBDF spent $1.1 million on research in 2014-2015. And, leveraged $698,000 more through partnerships and co-funding. Totaling almost $1.8 MILLION for Batten disease research this year.

Over the past seven years $15.5 MILLION has invested in research by leveraging donations, co-funding and partnerships. Leading to new avenues
for treatments or a cure and SIX POTENTIAL drug compounds for treatment. With a comprehensive strategy to advance all potential treatments and supporting projects, we are preparing for clinical trial success.

BBDF's research impact last year to this year -
Last year: 3 drug compounds under consideration.
This year: 6 drug compound options evaluated by industry partner Evotec and 2 lead candidates chose.
Last year: no pharmaceutical companies with programs in juvenile Batten disease.
This year: 3 companies with active programs or programs under development. Last year: no juvenile Batten human brain cell platform for discovery and drug testing.
This year: BBDF is funding the creation of the largest and most genetically diverse collection of induced pluripotent stem (iPS) cells in juvenile Batten disease. iPS cells are produced by reprogramming skin cells into brain cells. In other words, iPS cells will give researchers the first opportunity to watch juvenile Batten disease develop directly in human brain cells.
Last year: 1 promising approach to treating juvenile Batten disease.
This year: 4 promising approaches for treatment and possibly a cure.

In October 2014, Beyond Batten Disease Foundation and the New York Academy of Sciences brought together over 55 research leaders from multiple diseases, like Cancer, Parkinson’s and Alzheimer’s, to collaborate and focus their efforts and expertise on juvenile Batten disease. At the “Drug Targeting the
Lysosome Workshop,” which was held at the NYAS Conference Center at the World Trade Center, both academic and pharmaceutical researchers discussed how to work together towards common goals. There were many action items resulting from the meeting as well as an accessible and educational podcast.

Leadership

BBDF is led by a 6 member board of directors, comprised of individuals representing areas of expertise from the following sectors: medicine, government, financial management, public relations, philanthropy and investment.  Our research program is directed by a Principal Scientist who currently manages a prioritized research portfolio, including 38 concurrent projects.  She is responsible for seeking partnerships and collaborations and utilizes outside experts to review and recommend proposals before presenting them to the board.

Ask Us About

  • Building research collaborations
  • Development of tools and resources
  • Evaluating organizational performance