FDA Slams Break On Fulcrum Therapeutics’ Sickle Cell Disease Study, Shares Plummet
The FDA verbally informed Fulcrum Therapeutics Inc (NASDAQ:FULC) that it has issued a full clinical hold regarding the Investigational New Drug (IND)…
‘New Phase 3 Data Presented At WORLDSymposium Reinforce Nexviazyme As Potential New Standard Of Care For All People Living With Late-Onset Pompe Disease’
New Phase 3 data presented at WORLDSymposium™ reinforce Nexviazyme® (avalglucosidase alfa) as potential new standard of care for all people living with
Additional Interim Data From Phase I/II Trial Of REGENXBIO’S RGX-111 For The Treatment Of Severe MPS I Presented At WORLDSymposium
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
Earlier, Sanofi Announced FDA Approved Once-Weekly Altuviiio, New Class of Factor VIII Therapy For Hemophilia A That Offers Significant Bleed Protection
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.
4D Molecular Discloses Updated Data From Currently Paused Fabry Disease Cardiomyopathy Study
4D Molecular Therapeutics Inc (NASDAQ:FDMT) announced updated interim safety and efficacy data from the two 4D-310 INGLAXA Phase 1/2 trials for…
Sangamo Therapeutics Announces Evidence Of Clinical Benefit In Phase 1/2 STAAR Study In Fabry Disease
- 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
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
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
Gilead Presents Proof-Of-Concept Data For Investigational Combination Regimen Of Lenacapavir With Broadly Neutralizing Antibodies As A Potential Twice-Yearly Approach For The Treatment Of HIV
– Study Demonstrates the Potential of Lenacapavir in Combination with Broadly Neutralizing HIV Antibodies Teropavimab and Zinlirvimab –
– Findings Support Further Evaluation of the Investigational
ProPhase Labs Announces Plans To Initiate Clinical Trial Of Equivir, Goal To Launch Commercially In Q4 2023
ProPhase Labs, Inc. (NASDAQ:PRPH) ("ProPhase"), a growth oriented and diversified diagnostics, genomics and biotech company, today announced that its wholly-owned subsidiary, ProPhase BioPharma, Inc. (PBIO), is