#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…

BiotechAnalysst's tweet image. 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

CheckOrphan's tweet image. 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


$MREO ongoing discussions. #Alvelestat #AATD

doepke_michel's tweet image. $MREO ongoing discussions. #Alvelestat #AATD

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

CheckOrphan's tweet image. 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

doepke_michel's tweet image. 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?

alpha1europe's tweet image. 🧩 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?
alpha1europe's tweet image. 🧩 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?
alpha1europe's tweet image. 🧩 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?

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

CheckOrphan's tweet image. 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

doepke_michel's tweet image. $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

alpha1europe's tweet image. 📢 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

CheckOrphan's tweet image. 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

CheckOrphan's tweet image. 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

$ARWR relevant. $TAK #AATD $KRYS $MREO

fazirsiran in AATD-LD #xRNAx overlooked program?

BertrandBio's tweet image. fazirsiran in AATD-LD #xRNAx
overlooked program?
BertrandBio's tweet image. fazirsiran in AATD-LD #xRNAx
overlooked program?
BertrandBio's tweet image. fazirsiran in AATD-LD #xRNAx
overlooked program?
BertrandBio's tweet image. fazirsiran in AATD-LD #xRNAx
overlooked program?


📢 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

alpha1europe's tweet image. 📢 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?

alpha1europe's tweet image. 🧩 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?
alpha1europe's tweet image. 🧩 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?
alpha1europe's tweet image. 🧩 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. 💙

alpha1europe's tweet image. 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. 💙
alpha1europe's tweet image. 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. 💙
alpha1europe's tweet image. 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

alpha1europe's tweet image. 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

alpha1europe's tweet image. 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
alpha1europe's tweet image. 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

alpha1europe's tweet image. 🟢 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
alpha1europe's tweet image. 🟢 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:

alpha1europe's tweet image. 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:
alpha1europe's tweet image. 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:
alpha1europe's tweet image. 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

AGA_Gastro's tweet image. 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

RareDisease_Adv's tweet image. ✈️ 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

alpha1europe's tweet image. 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
alpha1europe's tweet image. 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
alpha1europe's tweet image. 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

alpha1europe's tweet image. 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
alpha1europe's tweet image. 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
alpha1europe's tweet image. 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
alpha1europe's tweet image. 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

rda_aatd's tweet image. 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

rda_aatd's tweet image. 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

rda_aatd's tweet image. 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 🎂🙂

AlphaCopdDoc's tweet image. Marking #RareDiseaseDay by doing #aatd clinic @uhbtrust in a balloon & poster decorated department, with cake for those who wanted it 🎂🙂
AlphaCopdDoc's tweet image. 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

KorroBio's tweet image. 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!

AlphaCopdDoc's tweet image. 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 💪🏻

AlphaCopdDoc's tweet image. 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 💪🏻

Liver stiffness by elastography as a predictor of subsequent endpoints in PiZZ #aatd #EASLCongress

AlphaCopdDoc's tweet image. Liver stiffness by elastography as a predictor of subsequent endpoints in PiZZ #aatd #EASLCongress

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

KorroBio's tweet image. 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

KorroBio's tweet image. 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 🧵

AlphaCopdDoc's tweet image. 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

WaveLifeSci's tweet image. 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

AlphaCopdDoc's tweet image. 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

ขิงเก่งขิงไวต้องยกให้เค้าเลย🤭🤭 #เนเน#Nene郑乃馨 #AATD

donmadondong's tweet image. ขิงเก่งขิงไวต้องยกให้เค้าเลย🤭🤭
#เนเน่
#Nene郑乃馨 
#AATD

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…

AlphaCopdDoc's tweet image. 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…
AlphaCopdDoc's tweet image. 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!

WaveLifeSci's tweet image. 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!
WaveLifeSci's tweet image. 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

KorroBio's tweet image. 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

WaveLifeSci's tweet image. 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

Share4Rare's tweet image. 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/

AlphaCopdDoc's tweet image. 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 😋

AlphaCopdDoc's tweet image. 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 😋
AlphaCopdDoc's tweet image. 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

CheckOrphan's tweet image. 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!

AlphaCopdDoc's tweet image. 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!
AlphaCopdDoc's tweet image. 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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