RELEASE: RELEASE: AskBio Receives Orphan Drug Designation for AB-1003(2)

(Information sent by the signatory company).

RELEASE: RELEASE: AskBio Receives Orphan Drug Designation for AB-1003(2)

(Information sent by the signatory company)

-AskBio receives orphan drug designation from the European Commission through its EU-based subsidiary BrainVectis for AB-1003, a new investigational AAV gene therapy for the treatment of limb muscular dystrophy ( LGMD)

-- LGMD is a rare form of muscular dystrophy with no approved treatment --

RESEARCH TRIANGLE PARK, N.C., Feb. 28, 2023 /PRNewswire/ -- Asklepios BioPharmaceutical, Inc. (AskBio), an independently managed subsidiary of Bayer AG, today announced that the European Commission (EC) has granted designation of orphan drug to AB-1003 (also known as LION-101)* for the treatment of limb muscular dystrophy (LGMD). AB-1003 is a novel investigational adeno-associated virus (AAV)-based recombinant gene therapy currently being developed as a single intravenous (IV) infusion for the treatment of patients with LGMD type 2I/R9 (LGMD2I/R9 ), a disease subtype that affects 4.5 people per million worldwide, including more than 5,000 people in the EU and US. The EC decision, dated February 15, 2023, follows a positive opinion from the Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency (EMA) and was received through AskBio's EU affiliate, BrainVectis.

AB-1003 is being investigated in the US in a phase 1/2 multicenter study that will evaluate the safety, tolerability, and efficacy of a single intravenous infusion of gene therapy in adult subjects with genotypically confirmed LGMD2I/R9.

"The EC Orphan Drug Designation for AB-1003 is an important recognition of the unmet medical need in LGMD, which has no approved therapy," said Sheila Mikhail, AskBio Co-Founder and CEO. "The burden that this rare form of muscular dystrophy places on patients and their families is significant, and this decision supports our efforts to potentially bring a new therapeutic option to people in the EU living with type 2I/R9 of this devastating disease."

The EC grants orphan drug designation to medicines intended to treat a life-threatening or chronically debilitating disease that affects no more than five people in 10,000 in the EU, provided that there is no other satisfactory therapeutic option or that the medicine may entail significant benefit to those affected by a specific disease. This designation will provide special incentives in the EU, such as the possibility to qualify for support for protocols and possible exemptions or reductions of certain regulatory fees. Additionally, if the drug is approved for commercialization, this designation will provide 10 years of commercial exclusivity.

About limb muscular dystrophy (LGMD) and limb muscular dystrophy type 2I/R9 (LGMD2I/R9)

Limb muscular dystrophy (LGMD) is a group of diseases that cause progressive weakness and atrophy of the muscles of the arms and legs.1 The muscles most affected are those closest to the body (proximal muscles). ), specifically the muscles of the shoulders, upper arms, pelvic area, and thighs.1 The severity, age of onset, and characteristics of LGMD vary among the many subtypes of the disease and are often inconsistent, even within the same family.1 Signs and symptoms can first appear at any age and generally worsen over time, although in some cases they remain mild.1

Limb muscular dystrophy type 2I/R9 (LGMD2I/R9) is a form of LGMD and is caused by mutations in the FKRP gene.2 In LGMD2I/R9, signs and symptoms usually appear in late childhood and may include difficulty running and walking.2 Symptoms gradually worsen over time to significant disability, and affected individuals typically rely on a wheelchair for mobility approximately 23-26 years after onset.2 Currently, no There is no treatment that modifies the progression of the disease, and management is based on the signs and symptoms present in each individual. Visit the National Institutes of Health Medline Plus website for more information on LGMD and LGMD2I/R9.

About AskBioAsklepios BioPharmaceutical, Inc. (AskBio), a subsidiary of Bayer AG and independently managed, is a fully integrated gene therapy company dedicated to developing life-saving and life-changing medicines. The company maintains a portfolio of clinical programs in a number of neuromuscular, central nervous system, cardiovascular, and metabolic disease indications, with a clinical-stage pipeline that includes therapies for Pompe disease, Parkinson's disease, and heart failure. congestive heart. AskBio's gene therapy platform includes Pro10™, an industry-leading proprietary cell line manufacturing process, and an extensive library of capsids and promoters. With global headquarters in Research Triangle Park, North Carolina, and European headquarters in Edinburgh, Scotland, the company has generated hundreds of proprietary capsids and promoters, several of which have entered clinical trials. An early innovator in the field of gene therapy, the company employs more than 800 people in five countries and holds more than 750 patents in areas such as the production of AAVs and chimeric and self-complementary capsids. Learn more at or follow us on LinkedIn.

About Bayer Bayer is a global company with core competencies in the fields of nutrition and health sciences. Its products and services are designed to help people and the planet prosper, supporting efforts to overcome the great challenges posed by an aging and growing world population. Bayer is committed to driving sustainable development and generating a positive impact with its businesses. At the same time, the Group aspires to increase its earning power and create value through innovation and growth. The Bayer brand is synonymous with trust, reliability and quality throughout the world. In fiscal year 2021, the Group employed around 100,000 people and had sales of €44.1 billion. R&D expenses before special items amounted to €5.3 billion. For more information, visit

AskBio Forward-Looking Statements This press release contains "forward-looking statements." Any statement contained in this press release that is not historical fact may be considered a forward-looking statement. Words such as "believes", "anticipates", "plans", "expects", "will", "intends", "potential", "possible" and similar expressions are intended to identify forward-looking statements. These forward-looking statements include, but are not limited to, statements regarding AskBio's pipeline of development candidates, manufacturing technology, and process. These forward-looking statements involve risks and uncertainties, many of which are beyond AskBio's control. Known risks include, but are not limited to, the possibility that AskBio may not be able to execute its business plans and objectives, including meeting expected or planned regulatory milestones and deadlines, its reliance on third parties, clinical development plans, processes and plans to manufacture and market its candidate products due to various reasons, including the current COVID-19 pandemic, potential limitations of the company's financial and other resources, manufacturing constraints that cannot be foreseen or resolved timely, potential disagreements or other issues with our external collaborators and partners, and the opinions or decisions of regulatory, judicial or other bodies, such as the opinions and decisions of the United States Food and Drug Administration or the Office of United States Patents and Trademarks. Any of the above risks could materially and negatively affect AskBio's business and results. Undue reliance should not be placed on any forward-looking statements contained in this press release. AskBio does not undertake any obligation to publicly update its forward-looking statements based on events or circumstances after the date hereof.

* The European Commission orphan drug designation is for "serotype 9 adeno-associated viral vector expressing fukutin-related protein."

1. National Institutes of Health – Medline Plus: Limb-Girdle Muscular Dystrophy – Description. Acceso el 17 de febrero de 2021. Limb-girdle muscular dystrophy: MedlinePlus Genetics

2. National Institutes of Health — National Center of Advancing Translational Sciences: Genetic and Rare Diseases Information Center. Limb-Girdle Muscular Dystrophy Type 2I – Summary. Acceso el 17 de febrero de 2021. Limb-girdle muscular dystrophy type 2I | Genetic and Rare Diseases Information Center (GARD) – an NCATS Program (

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