- February 10, 2020
Trial Triumphs, Takeover Talk Boost Reata Pharmaceuticals
Warren Huff, Reata’s president and CEO, discussed the clinical progress of omaveloxolone and bardoxolone, as well as the company’s approach to drug development, before the stock surge in an exclusive interview with GEN Edge during the recent J.P. Morgan conference in San Francisco . . .
- February 6, 2020
A Potential Cure for Hepatitis B
Dr. Lawrence Blatt, CEO, Aligos Therapeutics, discusses his company’s research around antisense oligonucleotides as potential therapies for chronic hepatitis B and other diseases of the liver. Despite an available vaccine, the incidence of chronic hepatitis B (HBV) remains high — the largest prevalence of any chronic virus that causes morbidity and mortality — in some regions.
- February 5, 2020
Step Aside, CRISPR: RNA Editing is Taking Off
Thorsten Stafforst found his big break at the worst possible time. In 2012, his team at the University of Tübingen in Germany discovered that by linking enzymes to engineered strands of RNA, they could change the sequences of messenger RNA molecules in cells. In essence, they could rewrite the genome’s instructions en route to making proteins.
- February 5, 2020
Developing Therapeutics for Rare Pediatric Diseases with Nevan Elam Rezolute
Nevan Elam, J.D. CEO and co-founder Rezolute, Inc. talks about their work developing therapeutics for congenital hyperinsulinism, a rare pediatric disease and for diabetic macular edema, a vision-related complication of diabetes. With a background of working with technology businesses, Nevan reflects on lessons learned and differences in drivers of innovation in the computer and biotech worlds.
- February 4, 2020
Twitter Takeovers: Forging Stronger Connections With Patient Communities
Awareness days and months occur throughout the calendar year to generate a greater understanding about various diseases and help increase visibility about the unmet needs related to these conditions. For many advocacy groups, these activities drive awareness and highlight rare disease states or disorders with great unmet needs.
- February 4, 2020
How a blind Alberta carpenter partially regained his sight
Dean Scott could hardly see two years ago, but the Spirit River, Alta., carpenter says an experimental drug helped him regain some of his vision and added colour to his life. Scott, 45, first noticed problems with his eyes when he was a child and lost his peripheral vision.
- February 3, 2020
Could ‘young’ blood stop us getting old?
In August of last year, Alkahest reported the results from a six-month trial that saw 40 patients with mild to moderate Alzheimer’s disease infused with a proprietary human plasma “fraction”. It appeared to arrest their expected mental decline.
- January 31, 2020
Families of rare-disease patients take on drug development
Frustrated with the slow pace of drug discovery and desperate to help their loved ones, untrained family members take the work into their own hands by starting biopharma companies. But few such companies have been successful.
