Pipeline & Science

PRECLINICALPHASE 1/2PHASE 3APPROVED

pz-cel

(brand name ZEVASKYN®)
recessive dystrophic epidermolysis bullosa (RDEB)

APPROVED

To learn more, please visit ZEVASKYN.com

PSMA SIR-T™

(ABO-701) Advanced Prostate Cancer

PRECLINICAL

Metastatic prostate cancer remains a leading cause of cancer mortality, with more than 30,000 deaths annually in the U.S. despite multiple prior lines of therapy. PSMA-SIR-T™, a first-in-class engineered autologous T-cell therapy, has demonstrated in preclinical studies durable tumor control, specificity to PSMA, and a modest cytokine release profile: a favorable safety signal absent in current cell therapy approaches. Its purpose-built SIR-T™ design is completely new, engineered to overcome the core failures of cell therapy. To learn more about SIR-T™ click here to watch a webinar from Dr. Preet M. Chaudhary, MD PhD, Professor of Medicine and Chief of Jane Ann Nohl Division of Hematology and Center for the Study of Blood Diseases at University of Southern California (USC) Keck School of Medicine, Director of USC Blood and Marrow Transplant and Cell Therapy Program.

PRECLINICALPHASE 1/2PHASE 3APPROVED

UX111

Sanfilippo Syndrome Type A (MPS IIIA)
Ultragenyx

APPROVED

FAYUVI™ (rebisufligene etisparvovec-hopf), formerly UX111 | Partnered with Ultragenyx. FAYUVI is an FDA-approved AAV vector-based gene therapy for the treatment of neurologic manifestations of mucopolysaccharidosis type IIIA (MPS IIIA, Sanfilippo syndrome type A) in pediatric patients with preserved neurodevelopmental function. To learn more, please visit FAYUVI.com.

TSHA-102

Rett Syndrome
Taysha Gene Therapies

PHASE 3

TSHA-102 is a self-complementary intrathecally delivered AAV9 investigational gene transfer therapy in clinical evaluation for Rett syndrome. Designed as a one-time treatment, TSHA-102 aims to address the genetic root cause of the disease by delivering a functional form of MECP2 to cells in the CNS. TSHA-102 utilizes a novel miRNA-Responsive Auto-Regulatory Element (miRARE) technology designed to mediate levels of MECP2 in the CNS on a cell-by-cell basis without risk of overexpression.

Undisclosed (AAV204 capsid)

Range of prevalent and rare retinal diseases
Beacon Therapeutics

PRECLINICAL

AAV204 is a novel AAV capsid being evaluated for use in potential gene therapies for a range of prevalent and rare retinal diseases. AAV204 has been shown to achieve high macular and optic nerve transduction levels after para-retinal administration and to facilitate transduction of both the inner and outer retina after intravitreal administration in preclinical studies. Beacon Therapeutics holds a non-exclusive license to AAV204 from Abeona for use in potential ophthalmology gene therapies.

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