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We develop life-changing therapies, focusing on rare diseases and aging-associated diseases using our cutting-edge technologies.

ZSCAN4 and Telomere Biology Disorders
Our ZSCAN4 technology is currently in clinical trials to extend telomeres in patients with Telomere Biology Disorders, a rare disease with significant unmet need.
Bobcat® mRNA
Our Bobcat® mRNA technology uses a proprietary mRNA design to deliver large payloads such as full-length dystrophin, which is mutated in Duchenne muscular dystrophy.

ZSCAN4’s features were originally discovered at the National Institute on Aging (NIH) by our Chief Scientific Officer, Minoru Ko, and his team. ZSCAN4 extends the telomeres of stem cells through a telomerase-independent mechanism and potentially has additional therapeutic features.

Future Therapeutic Candidates

Future candidates using ZSCAN4 may address diseases such as myelodysplastic syndromes (MDS), idiopathic pulmonary fibrosis (IPF), and aging-related diseases.

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ZSCAN4’s features were originally discovered at the National Institute on Aging (NIH) by our Chief Scientific Officer, Minoru Ko, and his team. ZSCAN4 extends the telomeres of stem cells through a telomerase-independent mechanism and potentially has additional therapeutic features.

Future Therapeutic Candidates

Future candidates using ZSCAN4 may address diseases such as myelodysplastic syndromes (MDS), idiopathic pulmonary fibrosis (IPF), and aging-related diseases.

Telomere

A telomere is a structure made of DNA sequences and proteins that exists at the ends of chromosomes. They exist to protect genetic data, like the plastic tips of shoelaces.

Telomeres shorten naturally as you age. In Telomere Biology Disorders, a genetic mutation leads to accelerated shortening and short telomeres from birth.

ZSCAN4 elongates telomeres through a telomerase-independent mechanism, which we can use to treat Telomere Biology Disorders and other aging-related conditions.

ZSCAN4

Lead Indication

People with Telomere Biology Disorders have abnormally short telomeres, which are the protective end-caps of DNA. They are born with or develop short telomeres and have accelerated shortening, which can lead to serious conditions, such as bone marrow failure.

The existing treatment, allogeneic hematopoietic stem cell transplant (HSCT), has potential issues such as immune reactions and an increased cancer risk.

Our Phase I/II clinical trial (NCT04211714) is in progress at Cincinnati Children’s Hospital Medical Center to study ZSCAN4 used in an autologous cell therapy in TBDs.

Early clinical data published in NEJM Evidence demonstrated the first-ever successful sustained telomere elongation in two EXG-34217-treated patients with a Telomere Biology Disorder, with no treatment-related safety concerns observed over a 24-month and 5-month period after EXG-34217 infusion.

Existing gene therapy vectors face payload size limits limits (e.g., the adeno-associated virus (AAV) can only carry a maximum payload of up to 4.7 kb). The proprietary design of Bobcat® mRNA allows our mRNA to carry payloads over 13 kb on a single strand, opening the door to treating several target indications with an unmet need.

Future Therapeutic Candidates

Future therapeutic candidates using Bobcat® mRNA may address diseases involving other large genes.

Lead Indication

People with DMD have mutations in dystrophin (11 kb), the largest gene in the human genome, leading to progressive muscle degeneration. Due to dystrophin’s large size, DMD therapies currently under development focus on delivering or restoring an incomplete dystrophin.

Bobcat® mRNA can encode full-length, normal dystrophin in a single mRNA strand, which may lead to improved stability and efficacy. Preclinical data has demonstrated restoration of muscular function in DMD model mice with no safety findings associated with administration or treatment.

Life-changing therapies for rare diseases and aging-associated diseases

ZSCAN4

Research Research
Preclinical Preclinical
Phase I/II Phase I/II
Telomere Biology Disorders
EXG-34217

Granted FDA Orphan Drug, Rare Pediatric Disease, and Regenerative Medicine Advanced Therapy (RMAT) Designations

Undisclosed
Aging-Related Diseases

Bobcat® mRNA

Research Research
Preclinical Preclinical
Phase I/II Phase I/II
Duchenne Muscular Dystrophy
EXG-7001
Preclinical phase
Undisclosed
Research phase

Our team comprises determined and experienced leaders who have a history of building companies and seeing candidates through commercialization.

Aki Ko

Chief Executive Officer & Co-Founder, Board Chairman
Aki Ko headshot

Aki Ko has served as Chief Executive Officer and Chair of the Board of Directors since May 2017, when he co-founded the company. Previously, Mr. Ko served as Chief Operating Officer for Elixirgen, LLC, the parent company of Elixirgen Therapeutics, and President of Elixirgen Scientific, LLC, a provider of stem cell reagent kits and related services. In his previous roles, Mr. Ko was responsible for operations and business development at Elixirgen, LLC and Elixirgen Scientific. Mr. Ko earned a Bachelor of Science degree from the University of Virginia, where he cultivated his interest in biology.

Minoru Ko, MD, PhD

Chief Scientific Officer & Co-Founder,
Board Member
Minoru Ko headshot

Minoru S.H. Ko, MD, PhD has served as Chief Scientific Officer and as a member of the board of directors since May 2017 and was a co-founder of the company with Akihiro Ko. In addition, Dr. Ko is currently professor emeritus at Keio University School of Medicine and formerly Professor and Chair at the Department of Systems Medicine, Keio University School of Medicine. In 2012, Dr. Ko founded Elixirgen, LLC to develop novel products based on stem cell related technology. In 2016, Dr. Ko founded Elixirgen Scientific, LLC. Prior to forming Elixirgen, LLC, Dr. Ko worked as Senior Investigator (tenured) and Section Chief at the National Institute on Aging, a division of the National Institutes of Health. He also previously served as Assistant Professor and Associate Professor (tenured) at Wayne State University School of Medicine. Dr. Ko has published more than 150 peer-reviewed scientific articles and more than 20 other articles and book chapters, including a “Stem Cell Biology” chapter of Harrison’s Principles of Internal Medicine (17th, 18th, 19th editions). Dr. Ko brings more than 30 years of experience in stem cell biology, genetics, molecular and cell biology, and systems biology.

Joel Rogers, CPA

Chief Financial Officer
Joel Rogers headshot

Joel D. Rogers, CPA has served as Chief Financial Officer since December 2019. Mr. Rogers previously worked at Osiris Therapeutics, Inc. (NASDAQ: OSIR), which was acquired by Smith & Nephew in April 2019, as Chief Financial Officer from May 2018 to October 2019 and Corporate Controller from October 2017 to May 2018. Mr. Rogers served as Corporate Controller at ARC Group Worldwide, Inc. (NASDAQ: ARCW) from August 2014 to October 2017. Prior to ARC Group Worldwide, Inc., Mr. Rogers served in accounting management roles at DigitalGlobe, Inc. (NYSE: DGI), Allos Therapeutics, Inc. (NASDAQ: ALTH), and Flex LTD. (NASDAQ: FLEX). Mr. Rogers began his career as an auditor at Deloitte & Touche LLP. He holds a Bachelor of Arts (Accounting) from the University of Washington and a Master of Business Administration from Cornell University. He is a Certified Public Accountant (Colorado).

Rick Finnegan

Chief Operating Officer
Rick Finnegan headshot

Rick Finnegan has served as Interim Chief Operating Officer since September 2022. Mr. Finnegan previously worked at SAB Biotherapeutics (NASDAQ: SABS) as Chief Business Officer and Executive Vice President of Program Management from September 2018 to May 2022. Mr. Finnegan served as Senior Vice President of Operations and Program Management at rEVO Biologics from 2014 to 2018 and Executive Vice President of Global Strategic Accounts and Commercial Development at Syneos, (formerly inVentiv Health) from 2010 to 2013. Mr. Finnegan led the European Specialty Pharma division for Glenmark Pharmaceuticals where he was employed from 2008 to 2010. He served as Senior Vice President of Sales and Marketing from 2003 to 2006 for Critical Therapeutics and Vice President of New Product Marketing for Sanofi (formerly Genzyme). From 1989 to 2001 Mr Finnegan served in a range of U.S. and Global commercialization roles for Merck & Co. He holds a Bachelor of Science in Business Administration from the University of New Hampshire and a Master of Science in Management from Massachusetts Institute of Technology.

Takamitsu Fujiu, PhD, JD

General Counsel
Takamitsu Fujiu headshot

Takamitsu Fujiu has served as General Counsel since January 2022, after spending 2021 serving as a legal advisor. Before joining Elixirgen Therapeutics, Inc., Dr. Fujiu worked for the law firm of Morrison & Foerster LLP from 2001 to 2020, where he started as a patent agent and eventually became a partner in the patent group. Before joining the law firm, Dr. Fujiu was a research engineer with focus on material sciences and later became a manager at Nikon Corporation in Japan. Dr. Fujiu holds a Bachelor of Science (Chemistry) from the University of Tokyo, a PhD (Material Sciences) from the Pennsylvania State University and a JD from the Catholic University of America.

Moriah Nachbaur

Chief Business Officer
Moriah Nachbaur headshot

Moriah K. Nachbaur has served as Interim Chief Business Officer since September 2022. Prior to joining Elixirgen Therapeutics, Ms. Nachbaur was CEO of MKN Biotech, Inc., a biopharma consulting firm specializing in integrated product development and corporate strategy. From 2013 – 2018, Ms. Nachbaur held numerous roles at Coherus BioSciences, Inc., including Finance, Alliance Management, Business Development, and Product Development. As a Senior Vice President of Product Development, she served as a member of the Executive Committee and led Program Management and Regulatory Affairs.

From 2006 – 2013, Ms. Nachbaur held numerous roles at Genentech, Inc., where she gained cross-functional experience including R&D, Early Development, Supply Chain & Manufacturing, Pricing & Contracting, and Commercial Launch. From 2001 – 2005, Ms. Nachbaur worked as a laboratory intern at Mendel Biotechnology, Inc. She holds an A.B. in Biochemical Sciences from Harvard University.

Aki Ko

Chief Executive Officer & Co-Founder, Board Chairman

Aki Ko has served as Chief Executive Officer and Chair of the Board of Directors since May 2017, when he co-founded the company. Previously, Mr. Ko served as Chief Operating Officer for Elixirgen, LLC, the parent company of Elixirgen Therapeutics, and President of Elixirgen Scientific, LLC, a provider of stem cell reagent kits and related services. In his previous roles, Mr. Ko was responsible for operations and business development at Elixirgen, LLC and Elixirgen Scientific. Mr. Ko earned a Bachelor of Science degree from the University of Virginia, where he cultivated his interest in biology.

Minoru Ko, MD, PhD

Chief Scientific Officer & Co-Founder,
Board Member

Minoru S.H. Ko, MD, PhD has served as Chief Scientific Officer and as a member of the board of directors since May 2017 and was a co-founder of the company with Akihiro Ko. In addition, Dr. Ko is currently professor emeritus at Keio University School of Medicine and formerly Professor and Chair at the Department of Systems Medicine, Keio University School of Medicine. In 2012, Dr. Ko founded Elixirgen, LLC to develop novel products based on stem cell related technology. In 2016, Dr. Ko founded Elixirgen Scientific, LLC. Prior to forming Elixirgen, LLC, Dr. Ko worked as Senior Investigator (tenured) and Section Chief at the National Institute on Aging, a division of the National Institutes of Health. He also previously served as Assistant Professor and Associate Professor (tenured) at Wayne State University School of Medicine. Dr. Ko has published more than 150 peer-reviewed scientific articles and more than 20 other articles and book chapters, including a “Stem Cell Biology” chapter of Harrison’s Principles of Internal Medicine (17th, 18th, 19th editions). Dr. Ko brings more than 30 years of experience in stem cell biology, genetics, molecular and cell biology, and systems biology.

Shauna Callahan

Board Member
Shauna Callahan, CPA has served as a director for the company since March 2020. From 2019 to present, Ms. Callahan serves as the Chief Financial Officer for CampMinder, LLC. Ms. Callahan also serves as a director for Anchor Center for Blind Children. Her experience has centered around providing financial expertise and diligence to mid-sized businesses navigating high growth both organically and by acquisition. Ms. Callahan started her career in public accounting at Arthur Andersen in Denver, Colorado subsequently holding financial roles in a variety of public and private companies in industries including market research, telecommunications, distribution, and manufacturing. She graduated Summa Cum Laude from the University of Colorado with a Bachelor of Science in Business Administration and Accounting.

Hiroaki Kobayashi, MD

Board Member
Hiroaki Kobayashi, has served as a director for the Company since March 2020. Since August 2019, Dr. Kobayashi has served as a Principal at the University of Tokyo Capital Partners and from August 2018 to July 2019, Dr. Kobayashi served as Business Development Manager of Boston Scientific Japan. Dr. Kobayashi has also served as the auditor of ConciergeU, Inc. since June 2021. Dr. Kobayashi also serves on the board of directors of TXP Medical (from July 2020 to the present) and the board of directors of Metcela, Inc. (from December 2020 to the present). Dr. Kobayashi received his MD from the University of Tokyo in 2010 and his MBA from the University of Michigan in 2018.

Thomas J. White, PhD

Board Member
Thomas J. White, PhD has served as a director for the company since March 2020. From 2001-2011, Dr. White served as Senior Vice President of Research and Development and Chief Scientific Officer at Celera Corporation. His research at Celera involved the discovery of new genomic, expression and proteomic biomarkers and the development and FDA registration of molecular diagnostic products for cystic fibrosis, Fragile-X, HIV drug resistance, hepatitis C virus genotyping, thrombosis, as well as laboratory developed (CLIA) tests for complex common diseases (cardiovascular, stroke, autoimmunity, liver) and breast cancer. From 1989-2000, Dr. White worked as Senior Vice President of R&D for Roche Molecular Systems, a diagnostics division of Hoffmann-La Roche. From 1978-1988, Dr. White was employed at the biotechnology firm Cetus Corporation where he held the positions of Senior Vice President of Research and Associate Director of Research and Development. Dr. White received his B.A. in Chemistry from Johns Hopkins University and a Ph.D. in Biochemistry from the University of California at Berkeley, including graduate studies in the Pharmacology Department at the Johns Hopkins School of Medicine.

Jeffrey L. Cleland, PhD

Board Member
Dr. Cleland has 30 years of industry experience in research and development, including more than a decade at Genentech, Inc. His experience in startups includes major roles in obtaining more than $450 million in capital at stages from Series A through D and exit via IPO including over $300 million in capital raised as CEO. As the founding CEO of Versartis (VSAR), he led one of top biotech IPOs of all time. After Versartis, he led the Series B financing and clinical translation of novel Johns Hopkins University technology as CEO of Graybug Vision (GRAY). He held executive management positions at BaroFold, Novacea and Targesome, and has managed directly all aspects of drug development and late-stage research. While at Genentech, Dr. Cleland served in product development and manufacturing roles. He held important leadership roles in the successful approval of two drugs, Herceptin® and Nutropin Depot®, as well as in early work on Lucentis®, Avastin®, and Kadcyla®. He holds a BS in Chemical Engineering from the University of California, Davis and a PhD in Chemical Engineering from the Massachusetts Institute of Technology. Dr. Cleland has authored more than 100 articles and four books, and holds several issued patents. He serves on the Boards of BIO, Exicure, and Zylem and has advisory roles with small emerging biotechnology companies.
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