Загрузка видео...

Не удалось загрузить видео

Возникла проблема при загрузке этого видео. Это может быть связано с временными проблемами сети или видео может быть недоступно.

На главную

𝐀𝐒𝐆𝐂𝐓 𝟐𝟎𝟐𝟓: Physicians and scientists raced to make a bespoke gene editing therapy for an infant who urgently needed one in months - a first hand account by Kiran Musunuru. #ASGCT2025 Full video:

BiotechTV

11,821 subscribers

22,313 просмотров • 1 год назад •via X (Twitter)

Здоровье и велнес Наука и технологии Образование #ASGCT2025

Anya Rossi• Live Now

Private livecam show

Комментарии: 5

Фото профиля Brian Korb

Brian Korb1 год назад

@kiranmusunuru Great story and better than the version I read in the WSJ!

Фото профиля The Institute for Functional Medicine (IFM)

The Institute for Functional Medicine (IFM)1 год назад

Transform health with functional medicine! Join IFM in San Diego to explore the latest clinical research from the field.

Фото профиля Harvey J. Berger, M.D.

Harvey J. Berger, M.D.1 год назад

@kiranmusunuru Fantastic interview, Brad. So exciting.

Фото профиля BioN00b

BioN00b1 год назад

@kiranmusunuru Incredible story. These people are modern day heroes. Everyone needs to start reposting these videos. Eventually we might get some cut through to other communities and begin to get more people interested in biotech. This stuff is way more exciting than 99% of other news stories.

Фото профиля Bioengineering Bro

Bioengineering Bro1 год назад

@kiranmusunuru Excellent communicator this guy could be a ceo

Похожие видео

𝐁𝐈𝐎 𝐈𝐧𝐭𝐞𝐫𝐧𝐚𝐭𝐢𝐨𝐧𝐚𝐥 𝟐𝟎𝟐𝟓: Ocugen CEO Shankar Musunuri discusses the company's gene therapy platform for eye conditions - one of which is already in a pivotal, with another to start. #BIO2025 $OCGN Full video:

𝐁𝐈𝐎 𝐈𝐧𝐭𝐞𝐫𝐧𝐚𝐭𝐢𝐨𝐧𝐚𝐥 𝟐𝟎𝟐𝟓: Ocugen CEO Shankar Musunuri discusses the company's gene therapy platform for eye conditions - one of which is already in a pivotal, with another to start. #BIO2025 $OCGN Full video:

BiotechTV

13,873 просмотров • 1 год назад

𝐀𝐒𝐆𝐂𝐓 𝟐𝟎𝟐𝟔: Michael Torres isn't slowing down after selling his antibody-drug conjugate company to Eli Lilly. Here's his gene therapy play. Full video:

𝐀𝐒𝐆𝐂𝐓 𝟐𝟎𝟐𝟔: Michael Torres isn't slowing down after selling his antibody-drug conjugate company to Eli Lilly. Here's his gene therapy play. Full video:

BiotechTV

10,996,973 просмотров • 4 месяцев назад

“I don’t think it’s exaggerating to say that this is the future of medicine.” For years, Dr. Rebecca Ahrens-Nicklas treated babies with rare genetic diseases knowing palliative care was often the only option. In 2023, she partnered with gene-editing researcher Dr. Kiran Musunuru to try something new: using CRISPR to correct disease-causing mutations before the damage became irreversible. In 2025, doctors delivered the first successful personalized CRISPR therapy, pointing to a future where gene therapies are custom-built for patients who once had no options. See the full TIME100 Health list of leaders, scientists, doctors, and more here:

“I don’t think it’s exaggerating to say that this is the future of medicine.” For years, Dr. Rebecca Ahrens-Nicklas treated babies with rare genetic diseases knowing palliative care was often the only option. In 2023, she partnered with gene-editing researcher Dr. Kiran Musunuru to try something new: using CRISPR to correct disease-causing mutations before the damage became irreversible. In 2025, doctors delivered the first successful personalized CRISPR therapy, pointing to a future where gene therapies are custom-built for patients who once had no options. See the full TIME100 Health list of leaders, scientists, doctors, and more here:

TIME

19,885 просмотров • 7 месяцев назад

Rylae-Ann Poulin was among the first to receive gene therapy directly to her brain to treat a rare genetic disease, an approach scientists say holds promise for treating a host of brain disorders. Reporting by Laura Ungar and video by Shelby Lum:

Rylae-Ann Poulin was among the first to receive gene therapy directly to her brain to treat a rare genetic disease, an approach scientists say holds promise for treating a host of brain disorders. Reporting by Laura Ungar and video by Shelby Lum:

The Associated Press

127,782 просмотров • 3 лет назад

A video shows improvements in hearing in a child who received gene therapy for 𝘖𝘛𝘖𝘍-related deafness. Full video and accompanying article:

A video shows improvements in hearing in a child who received gene therapy for 𝘖𝘛𝘖𝘍-related deafness. Full video and accompanying article:

NEJM

20,698 просмотров • 6 месяцев назад

𝐀 𝐅𝐢𝐫𝐬𝐭𝐡𝐚𝐧𝐝 𝐀𝐜𝐜𝐨𝐮𝐧𝐭: Jimi Olaghere's life was changed by the CRISPR based gene therapy CASGEVY. He says there is not enough awareness of or access to these types of therapies in the sickle cell disease community. Full video:

𝐀 𝐅𝐢𝐫𝐬𝐭𝐡𝐚𝐧𝐝 𝐀𝐜𝐜𝐨𝐮𝐧𝐭: Jimi Olaghere's life was changed by the CRISPR based gene therapy CASGEVY. He says there is not enough awareness of or access to these types of therapies in the sickle cell disease community. Full video:

BiotechTV

21,958 просмотров • 1 год назад

𝐂𝐨𝐦𝐩𝐚𝐧𝐲 𝐍𝐞𝐰𝐬: Solid Biosciences's CEO Bo Cumbo discusses today's data release of the company's microdystrophin gene therapy candidate for Duchenne muscular dystrophy. $SLDB Full video:

𝐂𝐨𝐦𝐩𝐚𝐧𝐲 𝐍𝐞𝐰𝐬: Solid Biosciences's CEO Bo Cumbo discusses today's data release of the company's microdystrophin gene therapy candidate for Duchenne muscular dystrophy. $SLDB Full video:

BiotechTV

42,193 просмотров • 1 год назад

IMO the most important scientific event this week was the publication of David R. Liu’s new Nature article 🧵👇in which he presented a new and promising Gene Editing modality named PERT - Prime Editing-mediated Readthrough of premature termination codons. This new modality - which uses Prime Editing to converted a regular tRNA gene into a highly optimized suppressor tRNA thus enabling permanent readthrough of premature stop codons, could potentially provide a one-time curative treatment for countless nonsense mutations which are responsible for almost one-third of all human genetic diseases! Here is an excellent lecture by Prof. David Liu - one of the most important scientists and innovators of our lifetime and the person who invented both Base editing and Prime editing - the 2 top leading modalities of Gene Editing, in which he throughly explains this huge scientific breakthrough. $PRME $BEAM

IMO the most important scientific event this week was the publication of David R. Liu’s new Nature article 🧵👇in which he presented a new and promising Gene Editing modality named PERT - Prime Editing-mediated Readthrough of premature termination codons. This new modality - which uses Prime Editing to converted a regular tRNA gene into a highly optimized suppressor tRNA thus enabling permanent readthrough of premature stop codons, could potentially provide a one-time curative treatment for countless nonsense mutations which are responsible for almost one-third of all human genetic diseases! Here is an excellent lecture by Prof. David Liu - one of the most important scientists and innovators of our lifetime and the person who invented both Base editing and Prime editing - the 2 top leading modalities of Gene Editing, in which he throughly explains this huge scientific breakthrough. $PRME $BEAM

Yair Einhorn

62,189 просмотров • 10 месяцев назад

The man who climbed Taipei 101, Alex Honnold, and Academy Award winner Anne Hathaway highlight the spirit of curiosity that drives scientific discovery. Reflecting on breakthroughs in gene editing, they honored the researchers and physicians behind life-changing therapies, including Kiran Musunuru, Rebecca Ahrens-Nicklas, Peter Marks, and 2025 Breakthrough Prize in Life Sciences laureate David Liu—whose work led to the treatment for baby KJ, also present at the event. Watch the 2026 Breakthrough Prize Ceremony premiere on Sunday, April 26 at 3 PM streaming on this platform.

The man who climbed Taipei 101, Alex Honnold, and Academy Award winner Anne Hathaway highlight the spirit of curiosity that drives scientific discovery. Reflecting on breakthroughs in gene editing, they honored the researchers and physicians behind life-changing therapies, including Kiran Musunuru, Rebecca Ahrens-Nicklas, Peter Marks, and 2025 Breakthrough Prize in Life Sciences laureate David Liu—whose work led to the treatment for baby KJ, also present at the event. Watch the 2026 Breakthrough Prize Ceremony premiere on Sunday, April 26 at 3 PM streaming on this platform.

Breakthrough

42,505 просмотров • 5 месяцев назад

𝐀𝐒𝐇 𝟐𝟎𝟐𝟓: KeloniaTx presented convincing late-breaking data at ASH today from its initial patients in multiple myeloma suggesting that in vivo CAR-T is on track to be a next innovation. Full video:

𝐀𝐒𝐇 𝟐𝟎𝟐𝟓: KeloniaTx presented convincing late-breaking data at ASH today from its initial patients in multiple myeloma suggesting that in vivo CAR-T is on track to be a next innovation. Full video:

BiotechTV

21,980 просмотров • 9 месяцев назад

𝐒𝐅 𝐇𝐞𝐚𝐥𝐭𝐡𝐜𝐚𝐫𝐞 𝐖𝐞𝐞𝐤: Sana Bio's CEO Steve Harr describes the science behind the company's cell therapy program that aims to be a cure for type 1 diabetes. $SANA Full video:

𝐒𝐅 𝐇𝐞𝐚𝐥𝐭𝐡𝐜𝐚𝐫𝐞 𝐖𝐞𝐞𝐤: Sana Bio's CEO Steve Harr describes the science behind the company's cell therapy program that aims to be a cure for type 1 diabetes. $SANA Full video:

BiotechTV

19,210 просмотров • 8 месяцев назад

This summer, the world learned that an infant -- baby KJ -- had been saved by gene editing. This morning at #STATSummit, we had KJ and his family on-stage. If you can watch this clip without tearing up... you may need a gene related to empathy edited. We're having an amazing day. Here are some memorable moments.

This summer, the world learned that an infant -- baby KJ -- had been saved by gene editing. This morning at #STATSummit, we had KJ and his family on-stage. If you can watch this clip without tearing up... you may need a gene related to empathy edited. We're having an amazing day. Here are some memorable moments.

Matthew Herper

35,988 просмотров • 11 месяцев назад

KABOOM‼️ 🚨 “CURES EXIST” Trump Announces FDA-Approved Gene Therapy Cure for Millions Born Deaf 🙌 The FDA has approved the first gene therapy for inherited deafness, a groundbreaking one-time treatment shown to restore hearing in children and others born profoundly deaf due to a rare genetic condition. Developed by Regeneron, Otarmeni, the therapy—will be made available at NO COST to eligible U.S. patients through a new Trump administration partnership—marks a historic medical breakthrough that could transform lives worldwide.

KABOOM‼️ 🚨 “CURES EXIST” Trump Announces FDA-Approved Gene Therapy Cure for Millions Born Deaf 🙌 The FDA has approved the first gene therapy for inherited deafness, a groundbreaking one-time treatment shown to restore hearing in children and others born profoundly deaf due to a rare genetic condition. Developed by Regeneron, Otarmeni, the therapy—will be made available at NO COST to eligible U.S. patients through a new Trump administration partnership—marks a historic medical breakthrough that could transform lives worldwide.

MJTruthUltra

156,444 просмотров • 5 месяцев назад

Congratulations to Jean Bennett, Katherine A. High and Albert Maguire, winners of the 2026 Breakthrough Prize in Life Sciences, for developing a therapy for inherited retinal degeneration that became the first FDA-approved gene therapy for a genetic disease. Penn Children's Hospital Rockefeller University

Congratulations to Jean Bennett, Katherine A. High and Albert Maguire, winners of the 2026 Breakthrough Prize in Life Sciences, for developing a therapy for inherited retinal degeneration that became the first FDA-approved gene therapy for a genetic disease. Penn Children's Hospital Rockefeller University

Breakthrough

22,438 просмотров • 5 месяцев назад

A video shows the administration of DB-OTO, an adeno-associated virus 1 gene therapy that delivers OTOF cDNA to treat congenital deafness. Watch the full video and read the accompanying article: #Otolaryngology

A video shows the administration of DB-OTO, an adeno-associated virus 1 gene therapy that delivers OTOF cDNA to treat congenital deafness. Watch the full video and read the accompanying article: #Otolaryngology

NEJM

37,122 просмотров • 6 месяцев назад

Tech + Tours: @gro_bio demonstrates how automated technologies have allowed its scientists to test wide spaces. Full video:

Tech + Tours: @gro_bio demonstrates how automated technologies have allowed its scientists to test wide spaces. Full video:

BiotechTV

17,042 просмотров • 3 лет назад

Company News: Intellia Therapeutics's Chief Scientific Officer elaborates on the news that the company successfully redosed patients with a CRISPR-based and LNP delivered therapy. $NTLA Full video:

Company News: Intellia Therapeutics's Chief Scientific Officer elaborates on the news that the company successfully redosed patients with a CRISPR-based and LNP delivered therapy. $NTLA Full video:

BiotechTV

21,950 просмотров • 2 лет назад