AskBio Inc. (AskBio)

AskBio is a leading clinical-stage gene therapy company dedicated to improving the lives of patients with rare diseases and other disorders.

[AskBio Inc. (AskBio) @ask_bio]

#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: https://t.co/yLocyD1m27

[AskBio Inc. (AskBio) @ask_bio]

#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 → https://t.co/wYIgr0W8au

[AskBio Inc. (AskBio) @ask_bio]

#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: https://t.co/xIoq2uTy5y

[AskBio Inc. (AskBio) @ask_bio]

#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 → https://t.co/Oe28qDhYdj

[AskBio Inc. (AskBio) @ask_bio]

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: https://t.co/6y6YSqUvbj

[AskBio Inc. (AskBio) @ask_bio]

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.

https://t.co/uF21gYFDAa

[AskBio Inc. (AskBio) @ask_bio]

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 → https://t.co/4VcrkqJ39i

#HeartFailure #NatureMedicine

[AskBio Inc. (AskBio) @ask_bio]

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: https://t.co/BSbgU9XPQZ

#AskBio #GeneTherapy

[AskBio Inc. (AskBio) @ask_bio]

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: https://t.co/arl2vgBfm8

[AskBio Inc. (AskBio) @ask_bio]

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.

https://t.co/IFhN9DjhhL

[AskBio Inc. (AskBio) @ask_bio]

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.

https://t.co/VoOUzwokLZ

[AskBio Inc. (AskBio) @ask_bio]

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 Inc. (AskBio) @ask_bio]

#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 👉https://t.co/uZDFk4I6Ol

#LGMD #BElieveTHEreisHOPEforacure #RareDisease

[AskBio Inc. (AskBio) @ask_bio]

#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: https://t.co/95JuHj39LF

#AskBio #Leadership #ParkinsonsDisease

[AskBio Inc. (AskBio) @ask_bio]

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.

https://t.co/aD8f9r9PV8

[AskBio Inc. (AskBio) @ask_bio]

#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

[AskBio Inc. (AskBio) @ask_bio]

At #ASGCT2025, #AskBio will share 15 presentations—including 3 orals—on advances in gene therapy, gene editing, and manufacturing.

For medical media: https://t.co/CXmjrtu8aR

[AskBio Inc. (AskBio) @ask_bio]

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: https://t.co/kx3NgIPtGb

[AskBio Inc. (AskBio) @ask_bio]

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

[AskBio Inc. (AskBio) @ask_bio]

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: https://t.co/gnSCatDoOr
#AskBio #Leadership