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A Mother's Mission - Project Baby Lion, ASO Therapy and the TNP02 Foundation with Yiwei She

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Manage episode 435552517 series 2918477
内容由Effie Parks提供。所有播客内容(包括剧集、图形和播客描述)均由 Effie Parks 或其播客平台合作伙伴直接上传和提供。如果您认为有人在未经您许可的情况下使用您的受版权保护的作品,您可以按照此处概述的流程进行操作https://zh.player.fm/legal

ONCE UPON A GENE - EPISODE 240

A Mother's Mission - Project Baby Lion, ASO Therapy and the TNP02 Foundation with Yiwei She

Yiwei She is a powerhouse mom to little Leo, the Founder of the TNP02 Foundation, a remarkable and brilliant advocate, and she's nothing short of extraordinary.

EPISODE HIGHLIGHTS

Can you tell us about Leo's diagnostic journey and how that inspired you to start the TNP02 Foundation?

Leo's head circumference wasn't what it should have been and then he had a seizure at two months old. Sequencing pointed to a mutation in a gene and we hit the ground running right away, tapping into our scientific backgrounds. At first, our world came crashing down, but we also had a very fortunate set of circumstances where we could maybe change Leo's outcome if we did the right things. We were thrown into the deep end, but we looked to others and learned a lot from the people who had already paved the way.

What is the process for developing an ASO therapy and how do you make the connections to launch it?

We had a friend that was in the biotech industry which proved to be a key link for us. We had other friends and parents that helped to connect us as well. Networking is really important because people can help guide you and advise on the best labs to work with.

Can you tell us about Project Baby Lion?

The idea behind Project Baby Lion is to take what I've learned and do it again better, in a more sustainable and systematic way. The first phase is to do diagnostics better, combining sustainability with urgency, starting with NICU whole genome sequencing. We hope to prove with data, the potential sustainability of personalized therapeutics within the ultra-rare populations. As early as possible, we want to connect patients and families with their advocacy groups, and where there isn't yet a community, we will try to put together a rapid program to develop a therapeutic plan.

What are the goals for Project Baby Lion and Leo's ASO therapy in the future?

The most helpful thing to come from Leo's trial will be the sharing of the data and clinical protocol so other families and foundations have a blueprint and recognize that it's possible. We want to put our data where our mouth is and share it out to catalyze a culture of sharing. For Project Baby Lion, I hope to attract investment and interest from the biomedical industry because we can solve the hard problems for our kids, and they're worth it.

LINKS & RESOURCES MENTIONED

TNP02 Foundation

https://www.tnpo2.org/

Project Baby Lion

https://www.tnpo2.org/project-baby-lion

Learn More and Register for Week in RARE

https://globalgenes.org/week-in-rare/

Creyon Bio

https://creyonbio.com/

Charles River Laboratory

https://www.criver.com/

Combined Brain

https://combinedbrain.org/

CONNECT WITH EFFIE PARKS

Website

https://effieparks.com/

Twitter

https://twitter.com/OnceUponAGene

Instagram

https://www.instagram.com/onceuponagene.podcast/?hl=en

Built Ford Tough Facebook Group

https://www.facebook.com/groups/1877643259173346/

  continue reading

321集单集

Artwork
icon分享
 
Manage episode 435552517 series 2918477
内容由Effie Parks提供。所有播客内容(包括剧集、图形和播客描述)均由 Effie Parks 或其播客平台合作伙伴直接上传和提供。如果您认为有人在未经您许可的情况下使用您的受版权保护的作品,您可以按照此处概述的流程进行操作https://zh.player.fm/legal

ONCE UPON A GENE - EPISODE 240

A Mother's Mission - Project Baby Lion, ASO Therapy and the TNP02 Foundation with Yiwei She

Yiwei She is a powerhouse mom to little Leo, the Founder of the TNP02 Foundation, a remarkable and brilliant advocate, and she's nothing short of extraordinary.

EPISODE HIGHLIGHTS

Can you tell us about Leo's diagnostic journey and how that inspired you to start the TNP02 Foundation?

Leo's head circumference wasn't what it should have been and then he had a seizure at two months old. Sequencing pointed to a mutation in a gene and we hit the ground running right away, tapping into our scientific backgrounds. At first, our world came crashing down, but we also had a very fortunate set of circumstances where we could maybe change Leo's outcome if we did the right things. We were thrown into the deep end, but we looked to others and learned a lot from the people who had already paved the way.

What is the process for developing an ASO therapy and how do you make the connections to launch it?

We had a friend that was in the biotech industry which proved to be a key link for us. We had other friends and parents that helped to connect us as well. Networking is really important because people can help guide you and advise on the best labs to work with.

Can you tell us about Project Baby Lion?

The idea behind Project Baby Lion is to take what I've learned and do it again better, in a more sustainable and systematic way. The first phase is to do diagnostics better, combining sustainability with urgency, starting with NICU whole genome sequencing. We hope to prove with data, the potential sustainability of personalized therapeutics within the ultra-rare populations. As early as possible, we want to connect patients and families with their advocacy groups, and where there isn't yet a community, we will try to put together a rapid program to develop a therapeutic plan.

What are the goals for Project Baby Lion and Leo's ASO therapy in the future?

The most helpful thing to come from Leo's trial will be the sharing of the data and clinical protocol so other families and foundations have a blueprint and recognize that it's possible. We want to put our data where our mouth is and share it out to catalyze a culture of sharing. For Project Baby Lion, I hope to attract investment and interest from the biomedical industry because we can solve the hard problems for our kids, and they're worth it.

LINKS & RESOURCES MENTIONED

TNP02 Foundation

https://www.tnpo2.org/

Project Baby Lion

https://www.tnpo2.org/project-baby-lion

Learn More and Register for Week in RARE

https://globalgenes.org/week-in-rare/

Creyon Bio

https://creyonbio.com/

Charles River Laboratory

https://www.criver.com/

Combined Brain

https://combinedbrain.org/

CONNECT WITH EFFIE PARKS

Website

https://effieparks.com/

Twitter

https://twitter.com/OnceUponAGene

Instagram

https://www.instagram.com/onceuponagene.podcast/?hl=en

Built Ford Tough Facebook Group

https://www.facebook.com/groups/1877643259173346/

  continue reading

321集单集

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