- | MeiraGTx
MEIRAGTX ANNOUNCES DATA FROM PHASE 1/2 CLINICAL STUDY OF GENE THERAPY FOR PATIENTS WITH X-LINKED RETINITIS PIGMENTOSA
MeiraGTx today announced top-line data from the Phase 1/2 clinical study MGT009 (NCT03252847) of botaretigene sparoparvovec (formerly referred to as AAV-RPGR), an investigational gene therapy in development for the treatment of patients with X-linked retinitis pigmentosa (XLRP) with disease-causing variants in the RPGR gene.
- | Arrowhead Pharmaceuticals
Drug reduces mutant protein that can lead to fibrosis in rare genetic liver disease
The experimental Arrowhead Pharmaceuticals drug fazirsiran can reduce the accumulation of a mutant protein by 83% among people with alpha1-antitrypsin deficiency (AAT) disease, according to results from an open-label phase 2 trial involving 16 volunteers.
- | Selecta Biosciences
What do you consider the biggest challenge that the pharma/biopharma industry currently faces?
The biggest challenge that the pharma and biopharma industries currently face is unwanted immunogenicity.
- | Aadi Bioscience
IN WHICH ORPHAN SPACES OR UNMET MEDICAL NEEDS DO YOU THINK WE WILL SEE THE NEXT TREATMENT INNOVATIONS?
We see huge potential for growth in precision medicine, particularly precision oncology, in the near future.
- | Sernova
NEW DATA, FILM HIGHLIGHT ISLET CELL TRANSPLANTATION PROGRESS
New data and a new documentary called “The Human Trial” together illuminate the hard work, sacrifice, and slow, iterative progress in the long search for a biological cure for type 1 diabetes.
- | Rain Therapeutics
RAIN TARGETS NORMAL P53 EXPRESSION WITH POSSIBLE PAN-CANCER THERAPEUTIC
Rather than target mutated p53, Rain Therapeutics is looking at normal p53 expression as part of its novel mouse double minute 2 homolog (MDM2) inhibitor therapy. In the coming 12 months, Rain expects to have an interim readout for a Phase II study of its liposarcoma therapy, called milademetan (Rain-32), and a pivotal readout for its Phase III trial of the same compound.
- | BlueSphere Bio
BlueSphere: TCR discovery at scale
BlueSphere is taking a high-throughput, lower-cost approach to TCR and neoantigen discovery that it thinks will enable multi-targeted cell therapies that stem resistance.
- | Xalud Therapeutics
Developing DNA Gene Therapies to Treat Chronic Inflammatory Diseases with Dr. Diem Nguyen Xalud Therapeutics
Dr. Diem Nguyen is the CEO of Xalud Therapeutics, a DNA delivery gene therapy company striving to develop and deliver medicines for large, broad-based disease indications like chronic inflammatory diseases, particularly musculoskeletal diseases like osteoarthritis of the knee and as well as neurodegenerative diseases like MS and ALS. Earlier in her career, Diem was the global President of Pfizer, covering multiple therapeutic areas, and has joined Xalud because of their ground-breaking DNA delivery platform.
