What Is A Hack-A-Thon?
A hackathon is an event that brings together the world's top minds to solve complex problems in a short period of time. In healthcare hackathons, we focus on tackling challenges in healthcare. The participants come from diverse backgrounds and possess a wide range of knowledge and skills, and together they can use an interdisciplinary approach to provide healthcare solutions that are actionable and real. In the Hackathon to Cure Michael, the challenge is to propose a cure for children affected by a disease called SPG50 resulting from a genetic defect in the AP4M1 gene quickly and safely before it robs them of their mobility and minds. Our Hackathon is based on our desire to give our son a better life and a brighter future. (MIT Hacking Medicine Website)
How We Got Here?
Over the last 8 months the Pirovolakis family has scoured the earth to find a cure for their son Michael and the other children. As it stands they are working on a gene replacement therapy through UT SouthWestern and drug screening through Boston Children's Hospital. We all know technology and advancements in treatments is changing daily, in an effort to leave no stone unturned we are hosting this amazing event.
But You Are Already Working On A Cure?
Unfortunately there is no guarantee that drug screening or gene therapy will result in a successful, quick or approved clinical trial. As such we are hoping you will help us find other potential treatment options such as (*RNA*, Antibodies, Nano-bodies, Protein Constructs, CRISPR, Zinc Fingers, Microbiome, Exosoxes, Small Molecules, Read-Throughs, ASO’s, and so many more..)
What Are Some Guidelines For This Hack-A-Thon
• Open to researchers and medical professionals
• Recommended team size of 4-6, various expertise recommended
• Must be translational in (18-24 Months)
• Must be realistic and with technology available today
• Must be a complete proposal accompanied by documentation and a video presentation
• It is recommended that proposals are in the form of a research grant request with detailed documentation on why a specific treatment option was chosen and how specifically it will work for SPG50 and or Michael's variants.
• Slack channels, Q&A and other support will be made available during challenge
Dates & Details
Kick-Off: Feb 22nd (Times See Below)
Challenge Closes: March 13th @5pm
Peer Review Opens: March 13th @9pm
Peer Review Closes: March 16th @9pm
Announcement Of Top 5: March 19th @5pm
Judging & End off Challenge: March 21st (12pm Est) Link To WebCast
*All times are Est & subject to change
Toronto: 12pm-4pm: Amazon Office - (120 Bremner Boulevard, 26th Floor)
Boston: 12pm-4pm: Amazon Office BOS17 - (27 Melcher St)
Washington: 12pm-4pm: Amazon Office WAS15 - (1800 South Bell St, Arlington, VA)
San Francisco: 9am-1pm: : Amazon Office SJC18 - (2100 University Ave, East Palo Alto)
London: 5pm-9pm: Amazon Office - (1 Principal Place, Worship St, London)
Barcelona: 5pm-9pm: Novartis NeuroHouse - (NeuroHouse: Passeig d´isabel II, 14, floor Principal Primera. Barcelona)
1st Prize: TBD (But working on something cool)
2nd Prize: $1500
3rd Prize: $500
Potential for a $25K Ontario Genomics grant for the best Ontario based team with an innovative idea.
We have created an open and accessible slack channel for you to collaborate with others, help form teams, answer questions and chat with mentors. Please use the link below to join the channel. Slack Channel Link Here
Due to the complexity of the problem and nature of an open, worldwide hackathon, teams are free to work on their own during the entire event. Teams are encouraged to attend if possible one of the kick-off locations but that is not mandatory or a restriction.
Dr. Steven Grey
Associate Professor at UT Southwestern Medical Center. Dr. Gray's core research focus is to develop adeno-associated virus (AAV) gene transfer vector systems, for clinically-relevant global gene transfer to the central and peripheral nervous system.
Dr. Jennifer Hirst
Principal Research Associate, Robinson lab, Cambridge Institute for Medical Research. Dr. Hirst is a cell biologist who discovered the AP-4 and AP-5 adaptor complexes and has been studying functionality and the link with Hereditary Spastic Paraplegia.
Dr. Darius Ebrahimi-Fakhari
Works at the Department of Neurology at Boston Children's Hospital. His group does research in Neurology, Neuroscience, and Cell Biology. Current projects include "Development Of An In Vitro Model Of SPG47/50/51 & 52-Associated Hereditary Spastic Paraplegia
Dr. Stephen Capuzzi
Is an expert in the fields of chemical information and computational modeling. He is particularly interested in the use of computational methodologies to accelerate rare disease drug discovery efforts.
Dr. Craig Blackstone Is a senior Investigator, Cell Biology Section, National Institute of Neurological Disorders and Stroke, NIH. Dr. Blackstone's laboratory investigates the cellular and molecular mechanisms underlying hereditary movement disorders. Craig is one of the most prominent HSP researchers in the world.
Dr. Alexandria Davies
Is focused on studying the AP-4 adaptor complex using CRISPR CAS9 and other tools. Her PhD research has been focused on the function of the AP-4 complex, particularly the cellular consequences of losing AP-4 and how this may lead to the severe neuropathology associated with AP-4 deficiency
Is currently positioned as a postbaccalaureate fellow in the Organic Acid Research Section of the National Human Genome Research Institute. He has dedicated his young career to the advancement of translational research for rare diseases, including his own
Dr. Angelica D'Amore
is currently studying the genetics of HSP, in particular of AP4-HSP forms. During her PhD she developed a MO knock-down model for AP4S1 (SPG52) and now she is working on the generation of a AP4B1 (SPG47) CRISPR-Cas9 KO mutant in a zebrafish model at Boston Children’s Hospital.
Dr. Xin Chen
Is currently the lead geneticist working on the Gene Therapy for SPG50 out of UT SouthWestern Medical Centre. He is also one of the lead instructors under Dr. Steven Gray
is currently a consulting Global Project Manager at Ipsen Biopharmaceuticals. He has managed drug and medical device development/commercialization projects in Rare Genetic Diseases, Oncology, Neurology, and Ophthalmology.
Kai Zhang is a project manager at QIAGEN where he leads, drives and motivates cross-functional teams and works cross-organizationally to ensure that custom OEM projects are appropriately specified, planned, resourced, and delivered to domestic and international pharma & biotech customers
Want to be a mentor? Email us your details and why you would be a good fit!