A Study to Evaluate the Safety of LION-101 in Subjects With Genetic Confirmation of LGMD2I/R9 (Part1)

Open to people ages 18-65

1. Male and female subjects between the ages 18 and 65 years with clinical diagnosis of LGMD2I/R9 and confirmation of FKRP gene mutation.
2. Ability to ascend 4 stairs between 2.5 and 10 seconds.
3. Ability to walk/run 10 meters in < 30 seconds.
4. Able to understand and comply with all study procedures.
5. Sexually active females of childbearing potential and female and male partners of male subjects receiving LION-101 must use a barrier method of contraception for the first 6 months after dosing.

1. Significant cardiomyopathy as defined by echocardiogram (left ventricular ejection fraction <40%), evidence of conduction defect (increased PR and RR intervals, left bundle branch block and QTcF >480m/sec), NYHA Class 3 or 4 heart failure, or MRI gadolinium enhancement evidence of clinically important myocardial fibrosis.
2. Contraindication to MRI or hypersensitivity to contrast dyes, shellfish or iodine.
3. Implanted spinal rods, cardiac pacemaker or other implantation that would distort cardiac MRI images.
4. History of chronic liver disease (e.g. hepatitis, HIV, steatosis) or abnormal liver function (abnormal GGT and/or abnormal total/direct bilirubin and/or AST and ALT >2 ULN).
5. Abnormal renal function (GFR < 60 ml/min, using the MDRD equation).
6. Any life-threatening disease, including malignant neoplasms and medical history or malignant neoplasms within the past 5 years prior to screening (except basal and squamous cell skin cancer).
7. In the opinion of the investigator, a pre-existing medical condition that predisposes the subject to risks that outweighs the potential benefits.
8. Requirement for daytime ventilatory support.

9. Change in glucocorticosteroid treatment within 3 months prior to baseline visit.

   10. Exposure to another investigational drug within 3 months prior to study treatment or

   any previous treatment with gene therapy.

   11. Ongoing participation in any other therapeutic clinical trial. 12. Neutralizing antibody titer to AAV9 ≥ 1:5. 13. Female subjects who are pregnant, plan to become pregnant in the next 12 months, or

   breastfeeding.