AskBio Inc. (AskBio)

#AskBio announces completion of enrollment in the Phase 2 GenePHIT trial of AB-1002 for heart failure. The trial randomized 173 participants, marking a key milestone. Initial results are expected in 1H 2027. Full Release → askbio.com/completion-of-…

FDA accepted #AskBio’s IND for AB-1009, an investigational gene therapy for late-onset Pompe disease (LOPD). AB-1009 also received Fast Track and Orphan Drug designations, highlighting unmet need for people with LOPD. Read more: askbio.com/fda-acceptance…

AskBio’s investigational gene therapies for Parkinson’s (AB-1005) and congestive heart failure (AB-1002) have been granted Japan’s Pioneering Regenerative Medical Product designation (SAKIGAKE)—advancing potentially innovative treatments for patients. bit.ly/4a6LOA8

We are proud to share that #AskBio’s 12-month data from its Phase 1 trial of investigational AB-1002 have been published in @Nature Medicine, a leading peer-reviewed journal. Read the full Nature Medicine publication → bit.ly/49dYWCQ #HeartFailure #NatureMedicine

AskBio announces presentation of initial safety data from the investigational Phase 1/2 LION-CS101 study of AB-1003 for LGMD2I/R9 at #WMS2025. For Medical Media: bit.ly/3Wu6tG3 #AskBio #GeneTherapy

At #ESGCT2025, #AskBio & Viralgen are sharing 6 presentations—1 oral and 5 posters —on advances in AAV gene therapy and manufacturing technologies, expanding potential to transform the lives of people. Medical media: askbio.com/askbio-announc…

AskBio announces first European participants randomized in REGENERATE-PD, our Phase 2 trial of investigational gene therapy AB-1005 for moderate Parkinson’s disease. Sites in Poland, Germany and the UK are now active; U.S. enrollment continues. bit.ly/4mDrM2Q

AskBio has completed enrollment in REGENERATE MSA-101, our Phase 1 trial of investigational AB-1005 for multiple system atrophy–parkinsonian type (MSA-P). This milestone marks progress toward a potential new treatment option for people living with MSA-P. bit.ly/4nC3cjD

Huge thanks to the patients, caregivers, families and advocates who joined us to share insights and elevate patient voices at the 2025 International Limb Girdle Muscular Dystrophy Conference! #AskBio #LGMD #2025LGMDConference #BElieveTHEreisHOPEforacure #GeneTherapy #RareDisease

#AskBio is proud to sponsor the 2025 Int’l LGMD Conf. Join us July 18-21 to learn how we’re integrating patient insights into clinical trial design and advancing #GeneTherapy for LGMD2I/R9. Learn more 👉bit.ly/43QcWQK #LGMD #BElieveTHEreisHOPEforacure #RareDisease

#AskBio announces publication of complete results of Phase 1b trial of AB-1005, an investigational #GeneTherapy for the treatment of Parkinson’s disease, in Movement Disorders: …mentdisorders.onlinelibrary.wiley.com/doi/10.1002/md… #AskBio #Leadership #ParkinsonsDisease

Today @escardio in Belgrade, we presented 12-month data from #AskBio’s Phase 1 trial of AB-1002, an investigational gene therapy for NYHA Class III heart failure (non-ischemic etiology). Phase 2 GenePHIT is now enrolling in Canada, Europe, and the US. bit.ly/3EZHOEr

#MSA2025 was a great success! Our symposium on #GeneTherapy and MSA brought together patients, caregivers, and scientists to share insights. Big thanks to @MissionMSA & all who joined the convo on the future of MSA research and care. #PatientCare #Innovation

At #ASGCT2025, #AskBio will share 15 presentations—including 3 orals—on advances in gene therapy, gene editing, and manufacturing. For medical media: askbio.com/askbio-announc…

Join us at @MissionMSA’s 2025 International MSA Congress!  AskBio's symposium on #GeneTherapy and MSA: Exploring Innovation Through Science and Patient Perspectives will be held on Saturday, May 10 from 3:30-5:00pm..   Learn more about #MSA2025: bit.ly/3EgImoL

Until next time, #AANAM! Thanks to all the #neurology professionals for joining engaging discussions about the future of #GeneTherapy. @AANmember

We’re proud to welcome Dr. Laura Sepp-Lorenzino to AskBio’s Board of Directors! With 30+ years of leadership in biotech, her insights will be key as we advance our clinical and pre-clinical #GeneTherapy candidates. Learn more: bit.ly/4crLjQj #AskBio #Leadership

We’re live at #AANAM 2025! Join us at booth #640 to connect about our investigational gene therapy for neurological diseases and discuss the latest updates in the field. See you there! @AANmember #Neurology #GeneTherapy

#AskBio will be attending @AANmember 2025 Annual Meeting in San Diego, CA! Connect with us at booth #640 to learn about AAV gene therapy in neurological diseases. Program: bit.ly/4iGuBOR #AANAM #GeneTherapy

What a weekend at #ACC25! Huge thanks to all who joined us for insightful discussions & engaging sessions! #GeneTherapy #HeartFailure #CHF #ClinicalTrials