AskBio_AAvenger_Newsletter_Winter2021

The AAvenger ADENO-ASSOCIATED VIRUS (AAV) GENE THERAPY NEWS

WINTER 2021 | VOL.3

BAYER ACQUIRES ASKBIO TO BROADEN INNOVATION BASE AskBio to operate autonomously and on an arm’s-length basis Sheila Mikhail, JD, MBA Chief Executive Officer and Co-Founder

We have exciting news to share. On October 26th, Bayer AG and AskBio announced an agreement under which Bayer would acquire AskBio, and that transaction successfully closed on December 1, 2020. To preserve our entrepreneurial culture as an essential pillar for nurturing successful innovation, AskBio will continue to operate as an independent company on an arm’s-length basis.

“Our innovation in capsid re-engineering and promoter design, coupled with our scaled manufacturing processes, gives us the tools to develop gene therapy solutions for more people suffering from a wider spectrum of diseases not being adequately treated today,” said Jude Samulski, PhD, Chief Scientific Officer and co-founder of AskBio.

commercialization strength to support us as we discover, develop and gain regulatory approvals for genetic medicines that could help people around the world. We will have the opportunity to change health care on an even bigger scale than we could as AskBio alone.

“With Bayer’s worldwide reach and translational expertise, especially in pathway diseases, our combined cultures of scientific advancement and commitment to patients, along with the retention of AskBio’s independent structure, position us to expedite development of gene therapy for more patients who could benefit from them,” said Sheila Mikhail, CEO and cofounder of AskBio.

By combining with Bayer, we join a leading global pharmaceutical company with the financial and

MEET THE TEAM: DR. CANWEN JIANG – CHIEF DEVELOPMENT & MEDICAL OFFICER Dr. Jiang is a medical executive with more than 25 years’ experience leading clinical development in therapeutic areas including genetic disorders, ophthalmology, cardiovascular, metabolic and oncology.

He joined AskBio this fall, and his extensive background in medicine and pharmaceutical development will be a tremendous asset as we continue to evolve and progress our clinical programs.

We’re committed to developing life-changing genetic medicines for patients who urgently need treatment options. We look forward to beginning clinical trials for LGMD, MMA and multiple system atrophy in early- to mid-2021.

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TRANSLATIONAL MEDICINE AT ASKBIO The term “translational medicine” can mean a lot of different things to different people, but in a simple way, its mission is to support the development of new drugs to treat diseases. In other words, translational medicine “translates” scientific discoveries into candidates for new medicines. This is an essential part of drug development.

At AskBio, scientists are hard at work assessing potential new treatments and how they can be used safely to treat diseases. They help identify biological responses to experimental treatments and then create the studies that help us understand the activity and safety of these discoveries for patients. Sometimes they are able to find biological markers to suggest when patients may be at increased risk for adverse reactions to treatment, or those that indicate the treatment is more likely to succeed.

When the consent forms of clinical studies ask you to agree to donate some of your blood or

tissue samples for research, it is often this team that studies these samples to learn how the many variations in human genetics can help predict the success of the new experimental treatments in development. Those research samples are incredibly important, and translational scientists are grateful for these contributions from our patientcollaborators.

“Translational medicine at AskBio is the critical bridge between the invention of new technology and the development of experimental treatments that may help people with serious diseases. I am honored to lead a team of innovative, expert scientists who work hard to find safe and effective solutions for people in need” said Dr. David Favre, Vice President of Translational Medicine at AskBio.

David Favre, Vice President of Translational Medicine

ASKBIO LIMB-GIRDLE INTERVIEW – DAN POPE Dan Pope was diagnosed with a non-specific form of LGMD in 2004 and received a genetic confirmation of LGMDR9 in 2013 while visiting the University of Iowa. He is active on social media serving as an advocate for the LGMDR9 community and has been volunteering with the CureLGMD2i Foundation since 2018.

How big of an impact does LGMDR9 have on your life and activities of daily living?

Living with LGMDR9 affects every aspect of my daily life and presents the kinds of challenges that most people would never even think about.

Simply getting out of bed, bathed and dressed can be challenging. Although I am still ambulatory, I’ve had a few falls that have caused me to have a very real fear of falling and not being able to get up on my own. This forces me to have to carefully think about everything I do, everywhere I go and every step along the way. On the bright side, LGMDR9 forces me to think creatively and be open to new and innovative ways of doing things.

Have you ever participated in a clinical trial for LGMDR9?

Yes, I participated in a myostatin inhibitor (domagrozumab) clinical trial, which was the first clinical trial specifically for LGMDR9. Unfortunately, the drug was not shown to be effective and its development did not move forward.

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How important is it to you to find an effective treatment for LGMDR9?

There’s a very urgent and vital need to find an effective treatment for patients like myself and the rest of the LGMDR9 community. Due to the unique and progressive nature of LGMDR9, patients in the later stages of the disease often experience cardiac and respiratory issues that can be life threatening. Preventing, or altering, this outcome is obviously quite important to me. I wish more people knew of the challenges, difficulties and fears that go along with it so that they could better understand the urgent need to find a cure.

Tell me how you first got involved with the CureLGMD2i Foundation?

The CureLGMD2i Foundation was started by the Brazzo family, whom I first met while attending the annual Dystroglycanopathy Patient and Family Conference at the University of Iowa. After meeting them, and many others at the conference, I knew that I’d found my tribe and I wanted to get involved. Raising money for research and spreading LGMD awareness were two things I could definitely support, and in 2018 I began volunteering with CureLGMD2i.

Dan Pope

CLINICAL TRIAL UPDATES

Sudhanthira Shanmugam, Scientist II

LIMB-GIRDLE MUSCULAR DYSTROPHY 2I/R9 Enrolling for Our First Clinical Study this Spring Based on encouraging pre-clinical animal models, we are planning to conduct a clinical trial of a gene therapy, LION-101, for limb-girdle muscular dystrophy 2i/R9 starting in Spring 2021.

We are developing LION-101 as a potential treatment to make up for the damaged, non-functioning gene, FKRP. We provide a correct copy of the FKRP gene using the shell of a harmless virus that has had the viral DNA removed. The gene is delivered to the voluntary and heart muscles, where it is expected to begin working like a non-damaged FKRP gene.

To limit hospital visits during this time of COVID-19, we are going to arrange as many in-home visits as are possible after the initial treatment. Please visit our website at www.askbio.com/genetic-disease-focus/limb-girdle for updates as they become available.

POMPE DISEASE CLINICAL STUDY ClinicalTrials.gov Identifier: NCT03533673 We are currently enrolling adult subjects with late-onset Pompe disease (LOPD) to assess multiple doses of the gene ACTUS-101. The current standard of care for Pompe disease involves patients receiving an infusion of the enzyme they cannot make (enzyme replacement therapy or ERT). ACTUS-101 is designed to deliver a functioning copy of the GAA gene (malfunctioning in Pompe disease) to the liver of subject, so that their own production of GAA is restored to a level sufficient enough to no longer require ERT. Initial enrollment is complete, and we are expanding enrollment through a protocol amendment.

CONGESTIVE HEART FAILURE ClinicalTrials.gov Identifier: NCT04179643 We are actively enrolling patients with NYHA Class III heart failure to assess three doses of NAN-101. NAN-101 is a gene therapy that aims to halt the progression of congestive heart failure.

The primary objective of this study is to assess the safety of NAN-101 for the treatment of NYHA Class III heart failure, as well as assess the impact of this treatment on patient health as measured by changes in exercise capacity, heart function and other factors including quality of life.

For more information, please visit www.clinicaltrials.gov or email us at askfirst@askbio.com.

Dr. Josh Grieger, Chief Technology Officer

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HOPE TRAVELS: CALLING ALL POMPE SUPERHEROES! Hope the Sloth has been adopted as the unofficial mascot of the Pompe community. In addition to studying gene therapy at AskBio, Hope is helping to fight crime and celebrate Halloween with her big brother Alfie as The AAVenger Superheroes! As Hope and Alfie continue to travel throughout our labs and the world, look forward to more updates!

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