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

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 THE 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)

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

Registration form -

https://app.smartsheet.com/b/form/ed0e509a9c1d411395128614f85932fb

Visit the Challenge website to see details of the challenge -

https://www.freelancer.com/nih/targeted-challenge

Q&A document -

https://app.box.com/s/b9zlzlz5lrbgnzcdhgckhuht1is8l0s1

Webinar recording -

https://youtu.be/J1s3O-pJAO0

August 24 Webinar #2 recording -

https://app.box.com/s/k60i5nxie3zt25ehzcqzh73y7yv9s9wc

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.

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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
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há 3 dias

Hi, We participated in the Phase 1 and we are trying to get a Unique Entity ID, but we don't have a Legal Business Name. Could you please let us know how we can proceed? Thanks.

há 2 meses

Hello Participants, The challenge is closed now! Thank you everyone for being a part of this. We will be announcing the winners prior to the Phase 2 launch - in December 2023. We will be making future updates on our website and Freelancer and NIH social media, stay tuned! If you have an questions please feel free to reach out to us via the Contact form on the Challenge Website: https://www.freelancer.com/nih/targeted-challenge

há 2 meses

Hello Participants, We are just a couple of hours away from the deadline! Make sure to submit your proposals on time. You can do it either via platform or via the Box, as was updated yesterday. If you have any questions, please do not hesitate to reach out to us at targeted_challenge@freelancer.com

há 2 meses

Anyone wants to build up a team for this contest?

há 3 meses

Hello Participants, Reminder: NIH Targeted Genome Editor Delivery (TARGETED) Challenge submission deadline is Thursday, October 5, 2023 at 5:00 PM ET The submission deadline is only 10 days away! All entries (submissions) must be completed and submitted by the deadline on Thursday, October 5, 2023 at 5:00 PM ET. We strongly encourage you to submit your entry as early as possible in order to meet this deadline. If you haven't registered yet, please make sure to do so soon, so our team can review and approve your eligibility; you will also receive any additional guidance and last minute updates. Please make sure to follow the Submission Guidelines and Challenge Announcement to ensure smooth submission (attached here in Files section). If you have questions as you are finalizing your submission, please reach out to us at targeted_challenge@freelancer.com or here on Clarification Board.

há 3 meses

check out #109 in which I have provided innovative ideas for the competition Hope it will be helpful to you.

há 3 meses

This is great contest...love it

há 4 meses

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.

há 4 meses

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

há 4 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