#aatd search results
YolTech Gene Editing Can Set a New Bar in #AATD, with >20uM AAT Levels at 45mg $BEAM $WVE $PRME $CRSP $KRRO businesswire.com/news/home/2026…
Tessera Therapeutics Announces FDA Clearance of IND Application for its Lead In Vivo Gene Editing Program TSRA-196 for Alpha-1 Antitrypsin Deficiency (AATD) - For More Information Visit shorturl.at/I7gez #AATD #Alpha1_Antitrypsin_Deficiency #Rare_Diseases @TesseraTx
Patients with AATD with #liver and/or lung disease face greater all-cause costs and healthcare resource utilization than patients with #AATD alone. Learn more: hcplive.com/view/cost-heal…
$REGN and Tessera Therapeutics to Jointly Develop TSRA-196, an Investigational Gene Editing Therapy for Alpha-1 Antitrypsin Deficiency (#AATD) globenewswire.com/news-release/2…
Attending the @AlphaFriend conference? Join us Saturday at 11:50am to learn about WVE-006, our first-in-class #RNAediting candidate being investigated in a clinical trial for people with #AATD. Register below (virtual or in person). #A1FNC24 #alpha1
Wave Life Sciences Announces Positive Update from Ongoing RestorAATion-2 Trial of WVE-006 in Alpha-1 Antitrypsin Deficiency - For More Information Visit shorturl.at/mRVPT @WaveLifeSci #Rare_diseases #AATD #OrphanDrugs #Drug_Research #FDA #Medical_Research
$WVE Announces First-Ever Therapeutic RNA Editing in Humans Achieved in RestorAATion-2 Trial of WVE-006 in Alpha-1 Antitrypsin Deficiency. #AATD $ARWR $MREO $KRYS $NTLA ir.wavelifesciences.com/news-releases/…
After $VRTX setbacks and the transaction between $INBX and $SNY, I believe that big pharma is looking for some #AATD assets. Huge opportunity - high unmet medical need. Some stocks to watch: $MREO $KRYS $ARWR $NTLA
🧩 Could unexplained skin marks be a missing piece of your health puzzle? They may signal #AATD Alpha-1 Antitrypsin Deficiency can appear as #Panniculitis — a rare skin condition causing painful red bumps, plaques or nodules due to protein breakdown. 🩺 Noticing these changes?
How to monitor #AATD lung disease - Delphi consensus. The latest collaborative effort from @EuroRespSoc EARCO group respiratory-research.biomedcentral.com/articles/10.11…
Sanofi’s Efdoralprin Alfa Earns Orphan Designation in the EU for Alpha-1 Antitrypsin Deficiency Related Emphysema - For More Information Visit shorturl.at/a59yW #AATD @sanofi #Rare_Diseases #OrphanDrugs
$BEAM Announces Positive Initial Data for BEAM-302 in the Phase 1/2 Trial in Alpha-1 Antitrypsin Deficiency (#AATD), Demonstrating First Ever Clinical Genetic Correction of a Disease-causing Mutation. investors.beamtx.com/news-releases/…
Really curious about what caused the shortfall in performance of the $krro 1st gen LNP-based #AATD candidate. Could it be in any way related to the underperformance of the $beam and $wve candidates in terms of M-AAT output relative to % transcript correction...and which I
𝐑𝐚𝐲𝐦𝐨𝐧𝐝 𝐉𝐚𝐦𝐞𝐬 𝐁𝐢𝐨𝐭𝐞𝐜𝐡 𝐈𝐧𝐧𝐨𝐯𝐚𝐭𝐢𝐨𝐧 𝐒𝐲𝐦𝐩𝐨𝐬𝐢𝐮𝐦: @KorroBio CEO @ramaiyar discusses how the company has chosen new RNA editing programs to move forward with this year. $KRRO Full video: biotechtv.com/post/korro-bio…
$KRYS get ready for busy months. KB408 #AATD interim readout by year end, KB707 first clinical data update in Q4. $WVE $MREO $NTLA
Backtrack to GalNAc: @KorroBio AATD strategy Rewrite post-fizzle bioworld.com/articles/726160 #biopharma #AATD #RNAediting
📢 Tomorrow at European Parliament 🇪🇺 “AATD awareness: progress & challenges” 22 Apr 10:00–12:00 CET. Hosted by MEP Chastel & A1EA. In context of European Alpha-1 Awareness Day (25 Apr). Watch live: lnkd.in/eRtn5mZD Join us! #Alpha1 #AATD
CRISPR Therapeutics to Present SyNTase Gene Editing Platform for Single-Dose In Vivo Correction of Alpha-1 Antitrypsin Deficiency - For More Information Visit shorturl.at/tWKPq @CRISPRTX #Rare_Diseases #AATD
A University of Birmingham (@unibirmingham) research shows how patients suffering from Alpha-1 antitrypsin deficiency ( #AATD ) are at higher risk of developing heart conditions - please check the link for more details ow.ly/suoB50R1eE3 #rarediseases #orphandrugs
📢 Tomorrow at European Parliament 🇪🇺 “AATD awareness: progress & challenges” 22 Apr 10:00–12:00 CET. Hosted by MEP Chastel & A1EA. In context of European Alpha-1 Awareness Day (25 Apr). Watch live: lnkd.in/eRtn5mZD Join us! #Alpha1 #AATD
🧩 Could unexplained skin marks be a missing piece of your health puzzle? They may signal #AATD Alpha-1 Antitrypsin Deficiency can appear as #Panniculitis — a rare skin condition causing painful red bumps, plaques or nodules due to protein breakdown. 🩺 Noticing these changes?
𝐖𝐨𝐫𝐥𝐝 𝐋𝐢𝐯𝐞𝐫 𝐃𝐚𝐲: AATD & Liver Health Alpha-1 Antitrypsin Deficiency (#AATD) can affect the liver at any age—often silently. Watch for signs like jaundice in infants or unexplained liver disease in adults. Early testing saves lives. Talk to your doctor. 💙
Exploring #AATD & liver health with Dr. Williams JH Griffiths 🏥 Hepatologist specialised in inherited liver disease & transplantation, sharing vital insights for better care. #RareDiseases #LiverHealth #Alpha1Europe #UnitedforAlpha1
Genes control how every cell in the body works. Some genes decide basic traits, such as eye/hair color. Other genes can increase the risk of getting certain diseases, #AATD alpha-1-antitrypsin #AAT #A1AT protein. #svpol Swede don´t get medicin mayoclinic.org/diseases-condi… @MayoClinic
Insightful session on #AATD & Bronchiectasis with Dr. Luciano Corda, founding member of Alpha1 Italy 🇮🇹 & expert pulmonologist 🫁 Sharing knowledge to improve patient care across Europe. #Bronchiectasis #LungHealth #RareDiseases #Alpha1Europe
🟢 Live from Warsaw 🇵🇱 ! The Alpha-1 Europe Alliance AGA is officially underway! 🌍✨ Excited to welcome all our member organisations for a day of collaboration, learning, and shaping the future of #AATD care across Europe. #Alpha1 #Alpha1Europe #PatientVoice #RareDiseases
Really curious about what caused the shortfall in performance of the $krro 1st gen LNP-based #AATD candidate. Could it be in any way related to the underperformance of the $beam and $wve candidates in terms of M-AAT output relative to % transcript correction...and which I
𝐑𝐚𝐲𝐦𝐨𝐧𝐝 𝐉𝐚𝐦𝐞𝐬 𝐁𝐢𝐨𝐭𝐞𝐜𝐡 𝐈𝐧𝐧𝐨𝐯𝐚𝐭𝐢𝐨𝐧 𝐒𝐲𝐦𝐩𝐨𝐬𝐢𝐮𝐦: @KorroBio CEO @ramaiyar discusses how the company has chosen new RNA editing programs to move forward with this year. $KRRO Full video: biotechtv.com/post/korro-bio…
Can persistent jaundice in a newborn be the missing piece? 🧩 It could point to Alpha-1 Antitrypsin Deficiency (#AATD) — a genetic condition where abnormal protein builds up in the liver, causing damage. Early signs matter. Diagnosis is simple: a blood test. Learn more:
Vikings mutation #AATD genetic inheritance alpha-1-antitrypsin deficiency: liver/lungs. #EU-Europe provides drugs, not #svpol! NT-rådet/@TLV_nyheter=1500 Swedes die prematurely=Denmark provides drugs facebook.com/groups/1254338… [email protected] alpha1awarenessday.org #alpha1
Vikings-mutation #AATD genetisk arv: alfa-1-antitrypsinbrist: lever/lungs. #EU-Europa ger läkemedel, inte #svpol! NT-rådet/@TLV_nyheter=1500 svenskar dör i förtid=Denmark ger läkemedlen @alpha1europe facebook.com/groups/1254338… [email protected] alpha1awarenessday.org #alpha1
Is there a 𝒎𝒊𝒔𝒔𝒊𝒏𝒈 𝒑𝒊𝒆𝒄𝒆 in our awareness of a crucial genetic condition?🧬 Alpha-1 Antitrypsin Deficiency (#AATD) is caused by mutations in the gene responsible for producing Alpha-1 Antitrypsin, a protein that helps protect the lungs and other tissues. When the
Important step. A structured algorithm using non-invasive tests and elastography can help identify AATD liver disease earlier. In practice, unexplained mild transaminitis with low platelets should prompt consideration and staged monitoring. #AATD #medx
International experts offer new consensus guidance for AATD-related liver disease including 1) An algorithm for screening, diagnosing & staging using non-invasive tests 2) A review of novel therapeutic approaches 3) Clinical trial criteria/endpoints. 🔗 ow.ly/CLYO50YEt20
✈️ Top Gun: Maverick and AATD – An Unexpected Connection Alyssa Gloor shares how Top Gun: Maverick surprisingly echoed her journey as a rare disease patient with #AATD. Read Alyssa's column: bit.ly/4t9OIur #RareDisease #PatientPerspective #TopGun #MedTwitter
Is there a 𝒎𝒊𝒔𝒔𝒊𝒏𝒈 𝒑𝒊𝒆𝒄𝒆 in your health puzzle affecting your breathing? 🧩 It could be Alpha-1 Antitrypsin Deficiency (#AATD)—a genetic condition that can cause lung damage, breathlessness & frequent infections. A simple blood test can help diagnose it. 🔗Learn
Is there a 𝒎𝒊𝒔𝒔𝒊𝒏𝒈 𝒑𝒊𝒆𝒄𝒆 in our awareness of a crucial genetic condition?🧬 Alpha-1 Antitrypsin Deficiency (#AATD) is caused by mutations in the gene responsible for producing Alpha-1 Antitrypsin, a protein that helps protect the lungs and other tissues. When the
This #WorldHealthDay, we highlight the importance of evidence-based decisions for the #AATD community 🔬 Trust science-based info to: • protect lung, liver & skin health • support early diagnosis • guide treatment decisions • improve quality of life Science drives earlier
Anyone with cirrhosis needs to get find out if they have AATD. This could mean better outcomes in patients with this condition. #AATD
March is national nutrition month and AATD patients can benefit from healthy diet. Generally the lower-inflammatory foods are best. #AATD #nationalnutritionmonth
The @US_FDA 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: bit.ly/4draiFV #RareDisease #YOLT202 # #Alpha1
AATD is a genetic condition that can affect even children of those with an abnormal alleles (such as PiZZ) in their genotype. It's largely under diagnosed. #AATD
Marking #RareDiseaseDay by doing #aatd clinic @uhbtrust in a balloon & poster decorated department, with cake for those who wanted it 🎂🙂
Attending Jones Virtual Genetic Medicine Day on Monday, November 25? Join us from 1:00–2:00 PM ET as our CEO & President, @ramaiyar, participates in a panel discussion focused on the role of RNA editing in Alpha-1 Antitrypsin Deficiency (#AATD). #RNAEditing #GeneticMedicine
Learning & networking at European #aatd masterclass. Hosted by pulmonology @MiravitllesMarc @CSLBehring but talking about liver too!
Great to learn from others in Canada & Scandinavia about approaches to accessing #aatd treatment. Lessons: understand the system & partner up to improve power and make change happen 💪🏻
Breaking news! The U.S. FDA has granted orphan drug designation to KRRO-110 an investigational medicine for the treatment of Alpha-1 Antitrypsin Deficiency (#AATD). The designation underscores the FDA's recognition of the growing need for new treatments for patients living with
Next week, our leadership team will be participating in a panel discussion at the @BMO Genetic Medicines Summit followed by @Piper_Sandler’s Virtual Two-Part Series featuring an AATD-focused fireside chat and expert webinar. $KRRO #AATD
Claims to be the most innovative square mile on the planet. Here to talk #aatd treatment using some exciting new technology @BeamTx press release in 🧵
Join us at 4:30 today at #ASGCT23 to learn about WVE-006, which is on track to be the first #RNAediting investigational therapeutic to enter clinical development. WVE-006 is uniquely designed to correct the mutation that causes #AATD and address both liver and lung disease. $WVE
Great to hear @CPathInstitute are working on generating endpoints for clinical trials in #aatd that are both meaningful to patients & accepted by regulators. Need both to get good treatment out there
Liver fibrosis occurs in ~25% #aatd patients & progresses slowly, possibly slower in the presence of #copd @GingerClarkMD @AATLDConference onlinelibrary.wiley.com/doi/full/10.11…
Over the weekend we met w/ the #AATD patient community at #A1FNC24 & liver specialists at the #EASLCongress to present on WVE-006, our best-in-class #RNAediting candidate for AATD. We remain on track for proof-of-mechanism in patients this year. Thanks for the great discussions!
Korro Bio is pleased to provide a pipeline update, and to announce the initiation of dosing in our REWRITE study investigating KRRO-110 as a potential treatment for Alpha-1 Antitrypsin Deficiency (#AATD). An interim readout is expected in the second half of 2025. Read the full
This morning we announced the submission of the first CTA for WVE-006. WVE-006 is the first-ever RNA editing program to enter clinical development and is designed to address #AATD-related lung disease, liver disease, or both. Read more: bit.ly/484uFU0. $WVE
We spoke with Mariano Pastor (@Alfa1Espana) about the importance of #respiratoryphysiotherapy in patients with #AATD. "Ideally, it should be done regularly." 📊 He also emphasizes: "Data is essential" to improve access to rehabilitation. 🔗 Read it here: tinyurl.com/2uecw58n
Exciting day talking #aatd trials with UK colleagues and @Kamada_Pharma More info 👉🏻 innovaate-study.com/en/home/
A pleasure to talk about #aatd in the rare disease symposium #BASL2024 Quick tour through diagnosis, liver disease progression & clinical trials. Lovely day for it too, topped off by a delicious slice of hazelnut cake before the journey home 😋
CRISPR Therapeutics Unveils Promising Preclinical Data for CTX460™, a SyNTase™ Gene Editing Therapy Targeting Alpha-1 Antitrypsin Deficiency - For More Information Visit shorturl.at/SEcfo @CRISPRTX #AATD #Rare_Diseases #OrphanDrugs #Alpha1_Antitrypsin_Deficiency
Great talk on exercise conceptualising how to treat limitations @Marian_Ramon. However #aatd evidence lacking v #copd and effects on muscle may be ⬇️. More research needed!
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