Rebisufligene etisparvovec

CGTLive®'s Weekly Rewind highlights 5 key developments in gene and cell therapies: Ultragenyx seeks FDA approval for MPSIII gene therapy UX111; Manali Kamdar discusses treatment sequencing for R/R LBCL; Arbor Biotechnologies' ABO-101 cleared for US trial in PH1; Tenaya's TN-201 shows early promise in HCM; EsoBiotec doses first patient with in vivo CAR-T ESO-T01 for multiple myeloma.

Ultragenyx Pharmaceutical Inc. (RARE) focuses on novel treatments for rare genetic diseases, with products like Crysvita and Mepsevii. It collaborates globally and reported a market cap of $4.031B, with a YTD return of 3.26% as of January 8, 2025.

Ultragenyx Pharmaceutical Inc., focusing on rare disease treatments, shows strong revenue growth and a promising pipeline, including a potential blockbuster for Osteogenesis Imperfecta. With a solid base business and significant market potential, its future hinges on clinical trial successes and regulatory approvals.

Ultragenyx Pharmaceutical submitted a BLA to the FDA for accelerated approval of UX111, a gene therapy for Sanfilippo syndrome type A. Supported by Phase 1/2/3 Transpher A trial data, UX111 reduces heparan sulfate levels, correlating with cognitive improvements. If approved, it would be the first U.S. therapy for this rare childhood dementia.

H.C. Wainwright maintains a Buy rating for Ultragenyx Pharma with a $95 target, citing its Phase 3 Aspire study for GTX-102 targeting Angelman syndrome. Ultragenyx shows strong revenue growth and liquidity, with a 42% increase in Q3 2024 revenue but a $134M net loss. The company aims for GAAP profitability by 2026, with potential new therapies on the horizon.

Ultragenyx submitted a BLA to the FDA for UX111 (ABO-102) AAV gene therapy for Sanfilippo syndrome type A (MPS IIIA) under the accelerated approval pathway. The BLA is supported by data showing UX111's ability to reduce heparan sulfate levels in the CSF and improve cognitive development. UX111 is well-tolerated and has various designations in the U.S. and EU. Ultragenyx also dosed the first patient in the Aspire study for Angelman syndrome.

Ultragenyx submitted a Biologics License Application (BLA) to the FDA for UX111, an AAV gene therapy targeting Sanfilippo syndrome type A. The BLA is supported by data showing UX111 reduces heparan sulfate in cerebral spinal fluid and correlates with improved cognitive development. UX111 aims to address the underlying enzyme deficiency causing the disease.

Ultragenyx submitted a Biologics License Application (BLA) to the FDA for UX111, an AAV gene therapy targeting Sanfilippo Syndrome Type A (MPS IIIA). If approved, UX111 would be the first treatment in the U.S. for this condition. The submission follows FDA's acceptance of CSF heparan sulfate as a surrogate endpoint for accelerated approval. Clinical data showed UX111 led to rapid, sustained decreases in CSF heparan sulfate levels, correlating with improved cognitive development. Most treatment-related adverse events were mild to moderate liver enzyme elevations that resolved.

Ultragenyx submitted a BLA to the FDA for UX111, a gene therapy for Sanfilippo Syndrome Type A, marking a potential first approved treatment in the U.S. UX111 targets the underlying enzyme deficiency, showing promise in reducing disease biomarkers and improving cognitive development.