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TARGETED (Targeted Genome Editor Delivery) Challenge: Phase 1 - Proposals

Open
Prêmio:
$75.000 USD
Inscrições Recebidas:
2
10 dias, 14 horas restante
Concurso marcado com sucesso!

Targeted Genome Editor Delivery (TARGETED) Challenge

Revolutionizing technology development of delivery systems for in vivo genome editing.

A $6,000,000 competition to improve in vivo delivery technologies for genome editors in two Target Areas: 1. Programmable delivery systems, and 2. Non-viral delivery across the blood-brain barrier.

YOU NEED TO FILL OUT REGISTRATION FORM AND BE REGISTERED ON THE FREELANCER.COM PLATFORM BEFORE SUBMITTING THE SOLUTION.

Subject of the Challenge

Gene editing holds the promise to treat genetic diseases at the source by correcting the faulty genetic patterns within our cells. The National Institutes of Health (NIH) has launched the TARGETED (Targeted Genome Editor Delivery) Challenge to advance genome editing technology by sourcing innovative solutions for delivering genome editors to somatic cells 
The Challenge is open to qualified groups or teams from organizations or institutions, particularly those in the genome editing or vehicle delivery fields, and will take place in three phases: Proposal, Preliminary Data, and Final Data, Independent Testing, and Validation.
The Challenge aims to improve the state of technology in two target areas:
Programmable Delivery System for Gene Editing. Solutions to Target Area 1 should be a highly efficient and programmable delivery system to deliver genome editing machinery that can target specific tissues or cell types. Solutions must have at least 3 configurations and be at least as efficient as the current state of the art.
Crossing the Blood Brain Barrier. Solutions to Target Area 2 must be highly efficient, non-viral delivery systems capable of crossing the BBB to deliver genome editing machinery to a substantial proportion of clinically relevant cell types in the brain.
The best solutions will progress to independent large animal testing and validation, with the potential to revolutionize gene editing technology and make a significant impact in treating genetic diseases.
The Challenge is a three-phase competition.
In Phase 1, Participants will be asked to submit a proposal describing their proposed solution and how it will address the requirements for one of the Target Areas. Participants may submit proposed solutions to both Target Areas but must do so with separate proposals that independently address each Target Area’s requirements. Up to ten proposals that are judged to best meet the requirements will each be awarded up to $75,000. Additional prizes of $50,000 may be awarded to additional meritorious solutions on the basis of the Judging Criteria.
In Phase 2, Participants must submit data from studies that demonstrate delivery and editing performance as well as describe their methodology, technology, and how their solution addresses the Challenge criteria. Participation in Phase 1 is not a requirement for participation in Phase 2; however, it is strongly encouraged. Up to 10 winners of Phase 2 will be each awarded $250,000 and will be eligible to compete in Phase 3. Only Phase 2 winners will be eligible to participate in Phase 3.
Phase 3 is separated into Phase 3a and 3b; all Participants must submit solutions for Phase 3a to be eligible to participate in Phase 3b.
For Phase 3a, Participants must submit all required information showing that their technology is ready for large animal testing through NIH-supported independent evaluation and has the ability to solve the requirements for one of the Target Areas. Up to 6 Participants will each be awarded $50,000 and will then prepare for reagent scale up and protocol development for NIH-supported large animal testing.
Participants who submit their reagents and protocols by the deadline for Phase 3b will have access to NIH-funded independent large animal testing to validate their solution. NIH will review the results and only award prizes to Participants whose solutions meet or exceed the criteria. The top successful solution in each Target Area will be awarded $625,000; the second place solution in each Target Area will be awarded $225,000; the third place solution in each Target Area will be publicly recognized and given an honorable mention award. Participants who participate successfully in all three phases could be awarded up to $1,000,000 in each Target Area.

Final (Phase 3) Solution Requirements

Target Area 1: Programmable Delivery System for Gene Editing 
Solutions must be a highly efficient and programmable delivery system to deliver genome editing machinery that can target specific tissues (cells, types, and/or organs). Solutions must be able to be programmed to deliver to at least three distinct and different cell(s), tissue types, and/or organs and with delivery and editing capability that is at least as efficient as the current state of the art. An optimal solution would be straightforward to manufacture, low-cost, scalable and have a reasonable safety profile. Solutions that propose viruses and viral-like systems or particles must build on the field and meet the criteria demonstrating full understanding of how the delivery system can be modified so that it is programmable and can target a variety of different tissue targets (cells, types, and/or organs). The solution will be judged on how well it meets the criteria.

Programmable solutions that only target central nervous system (CNS) targets should be submitted under Target Area 2. Solutions that meet the requirements for Target Area 2 but also are programmable to target a non-brain organ may be submitted for consideration in both Target Areas, though solutions submitted to both Target Areas are only eligible for one prize.
To be highly competitive in this Challenge, solutions must:  
Be programmable and target at least three distinct and different cell(s), tissue types, and/or organs.
Be able to deliver an editor and demonstrate delivery and editing that be at least as efficient as the current published efficiencies for the different tissue targets (cells, types, and/or organs) proposed.
Have a known biological mechanism for programmability: a clear relationship between what is done to modify the technology to deliver to different tissue targets (cells, types, and/or organs) and how this relates to the underlying biology and/or biochemistry of the system.   
Have conducted studies that demonstrate biodistribution and route of administration/delivery method.  
Demonstrate successful delivery and editing performance in large animals through NIH-supported independent evaluation. 
Have demonstrated a safety profile in experimental models consistent with other gene therapy/gene editing delivery systems intended for use in humans.
Solutions should also have these desired traits: 
Target more than one tissue/organ type. 
Be innovative in approach. 
 

Additionally, solutions could
Demonstrate market potential, competitive advantage, or potential to meet unmet medical needs.
Be able to be manufactured in a scalable and cost-effective manner.
 

Target Area 2: Crossing the Blood-Brain Barrier (BBB)
Solutions to Target Area 2 must be highly efficient, non-viral delivery systems capable of crossing the BBB to deliver genome editing machinery to a substantial proportion of clinically relevant cell types in the brain.

To be highly competitive in this Challenge, solutions must:  
Be able to traverse the BBB in vivo
Be able to deliver an editor and demonstrate delivery and editing in a substantial proportion of clinically relevant cell types in the brain. 
Be a non-viral delivery technology. Solutions may be virus-like particles and/or incorporate components of viruses in the proposed delivery technology. Solutions that are modifications of recombinant adeno-associated viral vectors do not meet this criterion.
Demonstrate successful delivery and editing performance in large animals through NIH-supported independent evaluation. 
Have demonstrated a safety profile in experimental models consistent with other gene therapy/gene editing delivery systems intended for use in humans.
Solutions should also have these desired traits:
Be innovative in approach. 
Be able to be manufactured, ideally synthetically, in a scalable and cost-effective manner.
This challenge is run under the America COMPETES Act.

IMPORTANT LINKS

Visit the Challenge website to see details of the challenge - https://www.freelancer.com/nih/targeted-challenge
Webinar recording - https://youtu.be/J1s3O-pJAO0
Please submit your questions in the challenge Public Clarification Board or to Targeted_Challenge@freelancer.com 
IMPORTANT: PLEASE READ THE FULL CHALLENGE ANNOUNCEMENT THAT CONTAINS THE CHALLENGE SUBMISSION REQUIREMENTS AND RULES.
Em Destaque Realçar Garantido Secreto Concurso Top

Habilidades Necessárias

Research Writing
Bioinformatics
Biology
Biomedical Engineering
Biotechnology
Chemical Engineering
Genetic Engineering
Medical
Medical Research
Microbiology

Formatos de Arquivos Aceitos

pdf

Quadro de Esclarecimentos
Não é permitido spam, autopromoção ou propaganda.

Avatar do Usuário
Moin Q.
·
há 5 dias
check out #109 in which I have provided innovative ideas for the competition Hope it will be helpful to you.
Avatar do Usuário
Rituparno M.
·
há 18 dias
This is great contest...love it
Avatar do Usuário
Organizador do Concurso
·
há 1 mês
Thanks to everyone for joining us this week for the webinar! We received a lot of questions and we are very excited to see your submissions. Recording can be accessed here: https://app.box.com/s/k60i5nxie3zt25ehzcqzh73y7yv9s9wc And the transcript of the Q&A was added to the Q&A tracker: https://app.box.com/s/b9zlzlz5lrbgnzcdhgckhuht1is8l0s1 Slides were uploaded here to the Files section.
Avatar do Usuário
Organizador do Concurso
·
há 1 mês
We are only 1 day away from our webinar/office hour with the NIH team. Make sure to register if you haven't already: https://freelancer.zoom.us/webinar/register/WN_WIskDH5CTviXi1-EDOWRBw
Avatar do Usuário
Organizador do Concurso
·
há 2 meses
Exciting news! We are hosting another webinar on August 24 at 12:00 PM EST for the National Institutes of Health TARGETED Challenge and you don't want to miss it! Join us as we answer all of your questions, as well as discuss the best way to approach this multi-phase challenge. Our panel of experts will share their insights and expertise on all of the above, with the hope of helping you submit the best solutions possible. This webinar is for anyone who is taking part or interested in taking part in the TARGETED Challenge. Register now to secure your spot: https://freelancer.zoom.us/webinar/register/WN_WIskDH5CTviXi1-EDOWRBw
Avatar do Usuário
Organizador do Concurso
·
há 2 meses
Hello everyone! We've updated the Q&A sheet once more, please feel free to review the updates: https://app.box.com/s/b9zlzlz5lrbgnzcdhgckhuht1is8l0s1
Avatar do Usuário
Boris S.
·
há 2 meses
It is not good to deceive the tax service.
Avatar do Usuário
Burhan U.
·
há 2 meses
Any entry from the US in this contest is very difficult! Because freelance is used more in Pakistan, Bangladesh and India, less in US etc
Avatar do Usuário
Sakeena B.
·
há 2 meses
wow, its amazing contests
Avatar do Usuário
Zain A.
·
há 2 meses
Any individual besides of having his team from any other country except U.S. residents can participate in this contest?

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