FDA Gives Clearance to In Vivo Gene-Editing Therapy for AATD The FDA has approved the Investigational New Drug application for YOLT-202, an in vivo gene-editing therapy for AATD.

The @fda.gov has approved the Investigational New Drug (#IND) application for YOLT-202, an in vivo gene-editing therapy for alpha-1 antitrypsin deficiency (#AATD), according to a recent press release.

Read more: https://bit.ly/4draiFV

#RareDisease #YOLT202 #GeneEditingTherapy #Alpha1

Gain insights on early multi-system clues in #AATD and how emerging RNA and gene-based therapies could shape future care. Jorge Lascano and Gerry McElvaney share guidance on coordinated lung–liver management and evolving treatment pathways

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Patient Groups Coalesce Under New 'United Liver' Banner to Tackle Rare Liver Diseases The stated mission of United Liver is to “transform lived experience into leadership, and collective action into measurable progress.”

The stated mission of United Liver is to “transform lived experience into leadership, and collective action into measurable progress.”

Read the full feature here: https://bit.ly/4kQ3ljf

#RareDisease #LiverDisease #AATD #MedSky

Empowering Clinicians through Innovative Rare Disease Education

🧬💻 @jacksonlab.bsky.social Clinical Education is proud to receive Alpha-1 Foundation funding to develop an AI-enhanced education program that helps future health care providers better diagnose & manage #AATD & other rare conditions.

2025 FDA/EMA Update on Orphan Drugs for AATD Emerging therapies for AATD may soon expand treatment options beyond lung manifestations to include liver and other organ involvement.

Emerging therapies for alpha-1 antitrypsin deficiency (#AATD), including gene therapies, RNA interference and small molecules, may soon expand treatment options beyond lung manifestations to include liver and other organ involvement @tarespirdis.bsky.social

Read now: https://bit.ly/4jVKRx6

Overcome challenges in diagnosing and managing #AATD #LiverDisease with Medthority. Earn up to 2.5 #CME credits now with accessible resources designed to suit you, whether you like to read, watch, or listen #MedSky #Hepatology #MedEd #LiverSky

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Quantitative CT of emphysema, wall thickness and mucus plugs in alpha-1-antitrypsin deficiency: relationship to clinical outcomes - European Radiology Objectives Alpha-1-antitrypsin deficiency (AATD) is a rare genetic disorder leading to chronic obstructive pulmonary disease (COPD). Emphysema is the major structural damage visible on CT scans.…

"✨ These findings highlight the clinical value of advanced #CT phenotyping for improved risk stratification and personalized management in AATD." - Sonja Jankovic

#ChestCT #QuantitativeImaging #AATD

buff.ly/2A7e53V (Gaël Dournes et al.)

Quantitative CT in alpha-1-antitrypsin deficiency (AATD) 🫁

This study demonstrates that AI-assisted #quantitative CT enables comprehensive assessment of #airway abnormalities beyond #emphysema in #AATD.

When should you consider genetic testing for AATD-associated #LiverDisease? Watch David Lomas address misconceptions in genetic testing, highlight complementary tools, and discuss future personalized therapies, available now on Medthority #MedSky #AATD #MedEd

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😮‍💨 How an #AATD nightmare became a valuable lesson…

AATD contributor Alyssa Gloor shares a recent scare: managing a cold and her rare lung disease—while realizing her inhaler hadn’t been refilled.

Read Alyssa's story here: https://bit.ly/3MybtaW

#RareDisease #RareVoices #PatientPerspective

Regeneron and Tessera ink $275 mil pact for gene editing therapy The companies will co-develop TSRA-196, a potential one-time genetic treatment for alpha-1 antitrypsin deficiency.

Regeneron Pharmaceuticals and Tessera Therapeutics sign a $275 million deal to develop TSRA-196, a potential one-time #GeneEditing therapy for Alpha-1 Antitrypsin Deficiency.

#News #deal #partneship #GeneTherapy #AATD #Pharma

Regeneron and Tessera ink $275 mil pact for gene editing therapy The companies will co-develop TSRA-196, a potential one-time genetic treatment for alpha-1 antitrypsin deficiency.

Regeneron Pharmaceuticals and Tessera Therapeutics sign a $275 million deal to develop TSRA-196, a potential one-time #GeneEditing therapy for Alpha-1 Antitrypsin Deficiency.

#News #deal #partneship #GeneTherapy #AATD #Pharma

#TLM25: Aleksander Krag, MD, PhD, gastroenterology professor of the University of Southern Denmark, speaking Nov. 8 about alpha-1 antitrypsin deficiency (#AATD) at @aasldnews.bsky.social 2025

Image & quote taken by Senior Correspondent, Larry Luxner.

#RareDisease #Hepatology #Alpha1 #MedSky

Alpha-1-Antitrypsinmangel verdient mehr Beachtung!
Eine Veranstaltung der Alpha-1-Center in Deutschland. Sichern Sie sich jetzt noch Ihre Teilnahme unter t1p.de/AATM2025
#alpha1antitrypsindeficiency #AATD #alpha1awareness #Alpha1AwarenessMonth

#UEGWeek kicks off this weekend! Stay up to date on the latest insights in gastroenterology with this accredited symposium on #AATD. Earn #CME credit now with expert advice on implementing multidisciplinary care and emerging therapies

#MedSky #UEGWeek2025

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Respiratory Weekly News – September 5th 2025 This Week in Respiratory Health: AATD Protein Breakthrough, Tyvaso IPF Win, CT:VQ FDA Nod and More From gene modulation in […] The post Respiratory Weekly News – September 5th 2025 appeared first on LucidQuest Ventures.

LucidQuest Views >>> Respiratory Weekly News – September 5th 2025 #News #AATD #COVID19 #CRSwNP #cysticfibrosis Comment below!

Alpha-1 Foundation Introduces AlphaDetect: A New Era in Disease Detection The Alpha-1 Foundation has launched AlphaDetect, a nonprofit aimed at revolutionizing detection methods for Alpha-1 Antitrypsin Deficiency, ensuring no patient is overlooked.

Alpha-1 Foundation Introduces AlphaDetect: A New Era in Disease Detection #USA #Durham #Alpha-1_Foundation #AlphaDetect #AATD

Respiratory Weekly News – August 8th 2025 Respiratory & Pulmonary Health Updates: mRNA Asthma Therapies, AI-Powered Wearables, IPF Breakthroughs & More This week’s Respiratory & Pulmonary roundup […] The post Respiratory Weekly News – August 8th 2025 appeared first on LucidQuest Ventures.

FYI: LucidQuest Views >>> Respiratory Weekly News – August 8th 2025 #News #AATD #AsthmaCare #cysticfibrosis #genetherapy Comment below!

Respiratory Weekly News – August 8th 2025 Respiratory & Pulmonary Health Updates: mRNA Asthma Therapies, AI-Powered Wearables, IPF Breakthroughs & More This week’s Respiratory & Pulmonary roundup […] The post Respiratory Weekly News – August 8th 2025 appeared first on LucidQuest Ventures.

ICYMI: LucidQuest Views >>> Respiratory Weekly News – August 8th 2025 #News #AATD #AsthmaCare #cysticfibrosis #genetherapy Comment below!

Respiratory Weekly News – August 8th 2025 Respiratory & Pulmonary Health Updates: mRNA Asthma Therapies, AI-Powered Wearables, IPF Breakthroughs & More This week’s Respiratory & Pulmonary roundup […] The post Respiratory Weekly News – August 8th 2025 appeared first on LucidQuest Ventures.

LucidQuest Views >>> Respiratory Weekly News – August 8th 2025 #News #AATD #AsthmaCare #cysticfibrosis #genetherapy Comment below!

What is #AATD, and how does it impact the liver and lung? Tune in to our accredited podcast series, Understanding AATD, now for insights from Pavel Strnad and Marc Miravitlles, plus listen to all 4 episodes to earn 1.5 #CME credits #MedSky #LiverSky

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Respiratory Weekly News – June 6th 2025 Lung & Respiratory Breakthroughs: STAT6 Degraders, IPF Cough Relief, and FDA-Cleared Respiratory Tech 🫁🚀 This week on Lung & Respiratory […] The post Respiratory Weekly News – June 6th 2025 appeared first on LucidQuest Ventures.

FYI: LucidQuest Views >>> Respiratory Weekly News – June 6th 2025 #News #AATD #asthmatreatment #clinicaltrials #COPD Comment below!

Respiratory Weekly News – June 6th 2025 Lung & Respiratory Breakthroughs: STAT6 Degraders, IPF Cough Relief, and FDA-Cleared Respiratory Tech 🫁🚀 This week on Lung & Respiratory […] The post Respiratory Weekly News – June 6th 2025 appeared first on LucidQuest Ventures.

ICYMI: LucidQuest Views >>> Respiratory Weekly News – June 6th 2025 #News #AATD #asthmatreatment #clinicaltrials #COPD Comment below!

Respiratory Weekly News – June 6th 2025 Lung & Respiratory Breakthroughs: STAT6 Degraders, IPF Cough Relief, and FDA-Cleared Respiratory Tech 🫁🚀 This week on Lung & Respiratory […] The post Respiratory Weekly News – June 6th 2025 appeared first on LucidQuest Ventures.

LucidQuest Views >>> Respiratory Weekly News – June 6th 2025 #News #AATD #asthmatreatment #clinicaltrials #COPD Comment below!

Also, #PrimeMedicine is in binding arbitration with #BeamTherapeutics in whether they can pursue #AATD under their agreement.

Beam Therapeutics Announces U.S. FDA Regenerative Medicine Advanced Therapy (RMAT) Designation Granted to BEAM-302 for the Treatment of Alpha-1 Antitrypsin Deficiency (AATD) CAMBRIDGE, Mass., May 12, 2025 (GLOBE NEWSWIRE) -- Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company developing precision genetic medicines through base editing, today announced that the ...

#FDA great news for all cell/gene Tx: #Beam gets RMAT designation for the #AATD base editor! Since they are focused on lung indication, it will likely be to facilitate BIOMARKER-based (serum alpha-1 levels) accelerated approval. finance.yahoo.com/news/beam-th...

Respiratory Weekly News – April 11th 2025 What’s New in Respiratory Space This Week Catch the latest lung and respiratory health headlines in under 5 minutes! From […] The post Respiratory Weekly News – April 11th 2025 appeared first on LucidQuest Ventures.

FYI: LucidQuest Views >>> Respiratory Weekly News – April 11th 2025 #News #AATD #LUNGHEALTH #RESPIRATORYRESEARCH Comment below!

Deep Visual Proteomics maps proteotoxicity in a genetic liver disease - Nature High-resolution spatial proteomics were used to map molecular events during hepatocyte stress in pseudotime across all fibrosis stages, recapitulating known disease progression markers and revealing e...

Further information on the study can be found here:

❕Publication: www.nature.com/articles/s41...
❕Press Release: www.biochem.mpg.de/alpha-1-anti...

#AATD #Alpha1Awareness @nature.com @frosenberger.bsky.social

Respiratory Weekly News – April 11th 2025 What’s New in Respiratory Space This Week Catch the latest lung and respiratory health headlines in under 5 minutes! From […] The post Respiratory Weekly News – April 11th 2025 appeared first on LucidQuest Ventures.

ICYMI: LucidQuest Views >>> Respiratory Weekly News – April 11th 2025 #News #AATD #LUNGHEALTH #RESPIRATORYRESEARCH Comment below!

Respiratory Weekly News – April 11th 2025 What’s New in Respiratory Space This Week Catch the latest lung and respiratory health headlines in under 5 minutes! From […] The post Respiratory Weekly News – April 11th 2025 appeared first on LucidQuest Ventures.

LucidQuest Views >>> Respiratory Weekly News – April 11th 2025 #News #AATD #LUNGHEALTH #RESPIRATORYRESEARCH Comment below!