Biotech - Spark Newswire

1 minute read

Orchard Therapeutics Announced Long-term Results From An Updated Integrated Analysis Of 39 Patients With Metachromatic Leukodystrophy Treated With Investigational OTL-200 In The Clinical Development Program

Today, 9:30 PM

Administration of one-time gene therapy resulted in statistically significant improvement in severe motor impairment-free survival with up to 11 years of follow-up (median 6.15 years) Composite primary endpoint used

ORTX

1 minute read

FDA
News

Earlier, Sanofi Announced FDA Approved Once-Weekly Altuviiio, New Class of Factor VIII Therapy For Hemophilia A That Offers Significant Bleed Protection

By Charles Gross

Today, 9:30 PM

The U.S. Food and Drug Administration (FDA) has approved ALTUVIIIO™ [Antihemophilic Factor (Recombinant), Fc-VWF-XTEN Fusion Protein-ehtl], previously referred to as efanesoctocog alfa, a first-in-class, high-sustained factor VIII replacement therapy.The U.S.

SNY

1 minute read

Roche Decides To Terminate Cancer Treatment Pact With Blueprint Medicines

By Vandana Singh

Today, 9:30 PM

Blueprint Medicines Corporation (NASDAQ:BPMC) will regain global commercialization and development rights to Gavreto (pralsetinib), excluding Greater China, following a…

BPMC

1 minute read

Sangamo Therapeutics Shares Updated Data From Fabry Gene Therapy Study, Says It Shows Evidence Of Clinical Benefit

By Vandana Singh

Today, 9:30 PM

Sangamo Therapeutics Inc (NASDAQ:SGMO) announced updated preliminary data as of October 20, 2022, the cutoff date from the Phase 1/2 STAAR…

SGMO

1 minute read

4D Molecular Discloses Updated Data From Currently Paused Fabry Disease Cardiomyopathy Study

By Vandana Singh

Today, 9:30 PM

4D Molecular Therapeutics Inc (NASDAQ:FDMT) announced updated interim safety and efficacy data from the two 4D-310 INGLAXA Phase 1/2 trials for…

FDMT

1 minute read

Denali Therapeutics Reveals New Interim Data From Early-Stage Hunter Syndrome Study

By Vandana Singh

Today, 9:30 PM

Denali Therapeutics Inc (NASDAQ:DNLI) announced new interim results from the ongoing open-label, single-arm Phase 1/2 study of DNL310 in children with…

DNLI

1 minute read

GSK, Pfizer And Shionogi Owned ViiV Healthcare Announced 12-month Findings From The Solar Phase 3B Head-to-head Study Of Long Acting Cabenuva Regime (CAB+RPV LA) Compared Against Gilead’s Biktarvy Regime (BIC/FTC/TAF)

By Benzinga Newsdesk

Today, 9:30 PM

Study findings showed that CAB+RPV LA dosed every two months achieved the primary endpoint of non-inferior virologic efficacy versus daily oral BIC/FTC/TAF, while 90% of participants who switched to CAB+RPV

GSK

6 minute read

Sangamo Therapeutics Announces Evidence Of Clinical Benefit In Phase 1/2 STAAR Study In Fabry Disease

By Happy Mohamed

Today, 9:30 PM

- Sustained, elevated expression of alpha-galactosidase A (α-Gal A) activity observed in 13 patients for over two years for the longest treated patient as of cutoff

SGMO

3 minute read

Additional Interim Data From Phase I/II/III CAMPSIITE Trial Of REGENXBIO’s RGX-121 For The Treatment Of MPS II (Hunter Syndrome) Presented At 19th Annual WORLDSymposium

By Happy Mohamed

Today, 9:30 PM

RGX-121, a potential one-time gene therapy for the treatment of MPS II, continues to be well-tolerated with no drug-related SAEs across three dose levels Additional data from patients in Cohort 3 using pivotal program

RGNX

1 minute read

Analyst Sees Attractive Entry Opportunity After BioCryst’s Pipeline Setback

By Vandana Singh

Today, 9:30 PM

Tucked in its Q4 earnings press release, BioCryst Pharmaceuticals Inc (NASDAQ:BCRX) reported a setback on the early-stage BCX10013 study. The company…

BCRX