Biotech - Spark Newswire

5 minute read

Additional Interim Data From Phase I/II Trial Of REGENXBIO’S RGX-111 For The Treatment Of Severe MPS I Presented At WORLDSymposium

Today, 6:22 PM

RGX-111 is an investigational AAV Therapeutic for the treatment of severe MPS I that is part of REGENXBIO's clinical-stage pipeline of neurodegenerative disease programs RGX-111, a potential one-time gene

RGNX

1 minute read

FDA Approves Sanofi’s Hemophilia A Therapy Offering Significant Bleed Protection

By Vandana Singh

Today, 6:22 PM

SNY

1 minute read

Why Cingulate Shares Are Trading Higher Today

By Vandana Singh

Today, 6:22 PM

Cingulate Inc (NASDAQ:CING) announced positive topline results from the CTx-1301-003 study assessing the effect of food on the absorption of its…

CING

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

By Benzinga Newsdesk

Today, 6:22 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, 6:22 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, 6:22 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, 6:22 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, 6:22 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, 6:22 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, 6:22 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