Clinical Research

Driving innovation in neurogenetics through observational studies and interventional trials of genetic therapies. 

Colin Quinn, MD
Colin Quinn, MD
PNTC Clinical Research Lead

The PNTC partners with both Penn investigators, academic partners, and biomedical companies to conduct observational studies and implement early-phase clinical trials of novel genetic therapies for patients with neurological disorders. The center brings together and amplifies clinical research expertise, leverages institutional resources, and streamlines internal startup operations to efficiently establish and conduct studies with the highest standards. Our team of investigators, operations specialists, and supporting clinical research staff provides focused, coordinated expert care and management. 

Clinical Trials & Research Studies

Activated Trials
Trial Sponsors
Patients Treated
Diseases Treated
Routes of Adminstration
Therapy Types
DNL-593

Phase: I / II

Therapy Type: Small Molecule

Indication: FTD

Sponsor: Denali Therapeutics Inc.

Principal Investigator: David Irwin, MD

Site Status: Active, Not Recruiting

Click here to access clinicaltrials.gov

LY3884961

Phase: I / II

Therapy Type: AAV Gene Therapy

Indication: FTD

Sponsor: Prevail Therapeutics

Principal Investigator: David Irwin, MD

Site Status: Active, Not Recruiting

Click here to access clinicaltrials.gov

AMT-260

Phase: I / II

Therapy Type: AAV Gene Therapy

Indication: MTLE

Sponsor: UniQure Biopharma B.V.

Principal Investigator: Michael Gelfand, MD, PhD 

Site Status: Active, Recruiting

Click here to access clinicaltrials.gov

SPK-10001

Phase: I / II

Therapy Type: AAV Gene Therapy

Indication: HD

Sponsor: Hoffmann-La Roche

Principal Investigator: Aaron Lasker, MD

Site Status: Active, Recruiting

Click here to access clinicaltrials.gov

AB-1005

Phase: II

Therapy Type: AAV Gene Therapy

Indication: PD

Sponsor: AskBio Inc.

Principal Investigator: Andres Deik, MD

Site Status: Active, Recruiting

Click here to access clinicaltrials.gov

AMT-162

Phase: I / II

Therapy Type: AAV Gene Therapy

Indication: ALS (SOD1)

Sponsor: UniQure Biopharma B.V.

Site PI: Colin Quinn, MD

Site Status: Active, Not Recruiting 

Click here to access clinicaltrials.gov

VTx-002

Phase: I / II

Therapy Type: AAV Gene Therapy

Indication: ALS

Sponsor: UniQure Biopharma B.V.

Site PI: Colin Quinn, MD

Site Status: Active

Click here to access clinicaltrials.gov

AOC 1001

Phase: III

Therapy Type: RNA Gene Therapy

Indication: DM1

Sponsor: Avidity Biosciences, Inc.

Site PI: Lauren Elman, MD

Site Status: Active, Not Recruiting

Click here to access clinicaltrials.gov

AOC 1001

Phase: III (OLE)

Therapy Type: RNA Gene Therapy

Indication: DM1

Sponsor: Avidity Biosciences, Inc.

Site PI: Lauren Elman, MD

Site Status: Active, Not Recruiting

Click here to access clinicaltrials.gov

VX-670

Phase: I / II

Therapy Type: RNA Gene Therapy

Indication: DM1

Sponsor: Vertex Pharmaceuticals Inc.

Site PI: Lauren Elman, MD

Site Status: Active, Not Recruiting

Click here to access clinicaltrials.gov

AOC 1020

Phase: II (OLE)

Therapy Type: RNA Gene Therapy

Indication: FSHD

Sponsor: Avidity Biosciences, Inc.

Site PI: Colin Quinn, MD

Site Status: Active, Not Recruiting

Click here to access clinicaltrials.gov

AOC 1020

Phase: III

Therapy Type: RNA Gene Therapy

Indication: FSHD

Sponsor: Avidity Biosciences, Inc.

Site PI: Colin Quinn, MD

Site Status: Active, Not Recruiting

Click here to access clinicaltrials.gov

AB-1009

Phase: I / II

Therapy Type: AAV Gene Therapy

Indication: LOPD

Sponsor: AskBio Inc.

Site PI: Paul McIntosh, MD

Site Status: Active, Recruiting

Click here to access clinicaltrials.gov

Descartes-08

Phase: III

Therapy Type: Cell Therapy

Indication: MG

Sponsor: Cartesian Therapeutics

Site PI: Chafic Karam, MD

Site Status: Active, Not Recruiting

Click here to access clinicaltrials.gov

BIIB058

Phase: III

Therapy Type: RNA Gene Therapy

Indication: SMA

Sponsor: Biogen

Site PI: Lauren Elman, MD

Site Status: Active, Not Recruiting

Click here to access clinicaltrials.gov

ALN-APP

Phase: II

Therapy Type: RNA Gene Therapy

Indication: CAA

Sponsor: Alnylam Pharmaceuticals

Principal Investigator: Aaron Rothstein, MD

Site Status: Active, Not Recruiting

Click here to access clinicaltrials.gov

BIIB105

Phase: I

Therapy Type: RNA Gene Therapy

Indication: ALS

Sponsor: Biogen

Principal Investigator: Lauren Elman, MD

Study Status: Closed

Click here to access clinicaltrials.gov

AOC 1020

Phase: I / II

Therapy Type: RNA Gene Therapy

Indication: FSHD

Sponsor: Avidity Biosciences

Site PI: Colin Quinn, MD

Site Status: Closed

Click here to access clinicaltrials.gov

PBFT02

Phase: I / II

Therapy Type: AAV Gene Therapy

Indication: FTD

Sponsor: PassageBio, Inc.

Principal Investigator: David Irwin, MD

Site Status: Closed

Click here to access clinicaltrials.gov

Branaplam

Phase: II

Therapy Type: Small Molecule

Indication: HD

Sponsor: Novartis Pharmaceuticals

Principal Investigator: Aaron Lasker, MD

Study Status: Closed

Click here to access clinicaltrials.gov

Descartes-08

Phase: II

Therapy Type: Cell Therapy

Indication: MG

Sponsor: Cartesian Therapeutics

Principal Investigator: Chafic Karam, MD

Study Status: Closed

Click here to access clinicaltrials.gov

BIIB094

Phase: I

Therapy Type: RNA Gene Therapy

Indication: PD

Sponsor: Biogen

Principal Investigator: Andrew Siderowf, MD

Study Status: Closed

Click here to access clinicaltrials.gov

LY3884963

Phase: I / II

Therapy Type: AAV Gene Therapy

Indication: PD (GBA1)

Sponsor: Prevail Therapeutics

Principal Investigator: Andres Deik, MD

Site Status: Closed

Click here to access clinicaltrials.gov

BIIB132

Phase: I

Therapy Type: RNA Gene Therapy

Indication: SCA3

Sponsor: Biogen

Principal Investigator: Ali Hamedani, MD

Study Status: Closed

Click here to access clinicaltrials.gov