KYSA-8: A Study of Anti-CD19 Chimeric Antigen Receptor T-Cell (CD-19 CAR T) Therapy, in Subject With Treatment Refractory Stiff Person Syndrome

Key Inclusion Criteria:

• Subject must have been diagnosed SPS per the following criteria:

• Rigidity of limb and axial (trunk) muscles prominent in the abdominal andthoracolumbar paraspinal areas and making bending difficult

• Clinical or electrophysiological evidence of continuous contraction of agonistand antagonist muscles

• Episodic spasms precipitated by unexpected noises, tactile stimuli, oremotional upset

• Absence of any other neurologic disease that could explain the stiffness andrigidity

• High titer serum anti-GAD65 antibodies shown at screening -OR- seropositive foranti-glycine antibodies. If anti-GAD65 antibodies are lower than the high titerthreshold peripherally but positive in the cerebrospinal fluid (CSF), thesubject can be included. A prior documented high titer anti-GAD65 antibodylevel may be acceptable subject to sponsor review.

• Active symptoms with inadequate response to at least one immunomodulatory therapy.

• Stiffness index ≥2.

• At least 20 of the 25 enrolled subjects should be ambulatory.

Key Exclusion Criteria:

• Bedridden subjects for more than 3 months.

• History of CNS or spinal cord tumor, metabolic or infectious cause of myelopathy,genetically inherited progressive CNS disorder, sarcoidosis, non-SPS progressiveneurologic condition or progressive multifocal leukoencephalopathy (PML).

• History of stroke, seizure, dementia, Parkinson's disease, cerebellar diseases,psychosis, aphasia, and any other neurologic disorder that is of a nature andseverity that the investigator considers would increase the risk for the subject.

• Cardiac ejection fraction ≤ 40%.