AskBio is a leading clinical-stage gene therapy company dedicated to improving the lives of patients with rare diseases and other disorders.
Research Triangle Park, NC
Joined May 2019
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#AskBio and our subsidiary @ViralgenVC are proud to announce 5 presentations at #ESGCT2026 in Hamburg, October 27–30. Our teams will highlight advancements in AAV manufacturing technologies for gene therapies.
Learn more: askbio.com/five-presentation…
GenePHIT, one of the largest gene therapy trials conducted in heart failure to date, successfully randomized more than 170 participants across 64 sites in 12 countries.
Baseline participant characteristics presented at #ESCCongress. Read more: bit.ly/4xwQmbR
@TCHheart
#AskBio announces dosing of the first participant in the Phase 1/2 PROGRESS-GT LOPD investigational clinical trial evaluating AB-1009 for late-onset Pompe disease, a rare and progressive genetic disorder.
Full announcement: askbio.com/first-participant…
#AskBio announces six presentations at the ASGCT 29th Annual Meeting, highlighting progress in gene therapy research and featuring advancements in manufacturing science from @ViralgenVC , our independently operated subsidiary.
Full Release → askbio.com/6-presentations-a…
#AskBio announces that it has introduced its proprietary, commercially ready manufacturing process to supply ametefgene parvec (AB-1005), an investigational gene therapy for the treatment of Parkinson’s disease and MSA-P.
Read More: askbio.com/viralgen-to-suppl…
#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-enr…
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-of…
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-announces-…
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: movementdisorders.onlinelibr…
#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-announces-…
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
