Astellas Gene Therapies

We’re looking forward to attending the Alliance for Regenerative Medicine’s Cell & Gene Meeting on the Mesa in Arizona! Learn more about the upcoming presentations from our Senior Vice President, Primary Focus Lead, Genetic Regulation, Richard Wilson, and Medical Head, Cell and Gene Therapy, Ha Tran, MD, MS below: #CGMesa24

On #InternationalAtaxiaAwarenessDay (IAAD), we shine a spotlight on #FriedreichAtaxia (FA) and the critical work being done to support this community. Hear from Christine Schlegel Brown, a champion for patient advocacy, on how she ensures the patient voice is central in developing #genetherapies at AGT. Join us in raising FA awareness and advocating for transformative treatments and patient care.

Myotonic dystrophy (#DM1) is caused by a genetic mutation preventing specific genes from functioning properly, impacting muscle systems throughout the body. People living with DM1 have a 50% chance of passing it on to their children. Early genetic testing and counseling can save lives – help raise awareness in the medical community by supporting #myotonicDystrophyAwareness Day & learn more below.

#DYK Myotonic Dystrophy Type 1 (#DM1) is an inherited form of muscular dystrophy affecting the muscles and other body systems? DM1 ranges in age of presentation from congenital, infantile, juvenile, and adult, as well as level of severity. Learn more about the symptoms of DM1: 

Astellas Gene Therapies recently participated at Friedreich's Ataxia Research Alliance (FARA)’s Research Reception in Illinois. The meeting enabled us to hear directly from the patients and families affected by Friedreich Ataxia (FA) and share Astellas’ commitment to investigating potential gene therapies for people with cardiomyopathy from FA. Our partnership with the FA community is building and growing as we continue to involve the patient voice in every step of drug development.

#ICYMI, check out the “Medical Stories” docuseries airing on PBS local stations that was sponsored by Astellas to share personal stories of families impacted by X-linked myotubular myopathy (#XLMTM) and to help raise awareness of the condition. https://bit.ly/4eYxIAE Disclaimer: This video was not created or produced by Astellas.

At AGT, the patient voice is integral to our innovation and decision-making process. We actively engage with patients and caregivers, using their insights to shape our protocols and clinical trial materials. By collaborating with patient advocacy groups and directly with individuals through our patient ambassador programs, we gain real-time, actionable feedback that drives our mission forward. Our Medical Head of Cell and Gene Therapy, Ha Tran, MD, MS, shares how this approach differentiates us:

Join us all at Astellas in welcoming our talented group of summer #interns! These bright minds are joining us from diverse backgrounds and educational fields spanning from health policy to biomedical engineering. We’re looking forward to the fresh perspectives they’ll bring to our team this year. Let's give a warm welcome to these future innovators!

Friedreich ataxia (#FA) is a rare, inherited disorder that often takes years to diagnose due to its complex and varied symptoms. At AGT, we understand the profound impact this diagnostic journey and disease has on patients and their families. We are committed to developing a #genetherapy that targets the root cause of FA. 

#DYK that #genetherapies aim to treat the underlying genetic cause of disease by introducing a new gene or editing genes directly? At AGT, we are investigating gene therapies for multiple rare diseases, including #PompeDisease, #FriedreichAtaxia, #MyotonicDystrophy Type 1, and #XLMTM. Learn more about the different approaches to gene therapy, dependent on the condition: https://lnkd.in/gemsEw2c