Biotech Companies continue to show resurgence even in these challenging times, with more and more companies persevering with bringing their clinical candidates to fruition. While Vaccine development and therapeutics to combat the COVID-19 pandemic are at the forefront of development activities in the biotech sector,...
Fabry Disease also known as angiokeratoma corporis diffusum or alpha-galactosidase A deficiency, is an inherited disorder characterized by the buildup of globotriaosylceramide, a type of fat in the body’s cells and results in numerous disorders such as acroparesthesias (burning, tingling, or prickling sensation in extremities),...
Catabasis Pharmaceuticals (NASDAQ: CATB), a clinical-stage Company leveraging its proprietary SMART (Safely Metabolized and Rationally Targeted) Linker drug discovery platform to treat multiple biological targets announced a partnership with Duchenne UK, a London-based charity that funds treatments for Duchenne Muscular Dystrophy (DMD). The partnership will...
Wave Life Sciences (NASDAQ: WVE), a clinical-stage Company developing novel stereopure oligonucleotides to treat genetic defects, announced topline data from an ongoing Phase 1b/2a PRECISION-HD2 trial evaluating the safety and efficacy of WVE-120102, a first-in-class allele-selective approach to treat Huntington’s disease (HD). The study shows...
According to an estimate by the Centers for Disease Control and Prevention (CDC), SCD affects over 100,000 Americans and is a major public health concern as it resulted in 75,000 hospitalizations between 1989 through 1993, and cost over $475 million. The Medical expenditure of children with...
CRISPR, which is an acronym for Clustered Regularly Interspaced Short Palindromic Repeats, is a revolutionary genome editing approach that helps change an individual’s DNA, by altering it at a specific location. The Crisper-Cas9, short for clustered regularly interspaced short palindromic repeats and CRISPR-associated protein 9,...
Sarepta Therapeutics (NASDAQ: SRPT) faced a setback recently with the FDA rejecting Vyondys 53 (Golodirsen), an experimental drug for Duchenne muscular dystrophy, which targets over 8% of the DMD patients with a rare genetic mutation. The company was issued a Complete Response Letter (CRL), which...
Earlier this year, we had covered some path-breaking companies engaged in clinical trials, to test the efficacy and safety of novel therapeutics, which could help in the treatment and management of Duchenne Muscular Dystrophy (DMD). Capricor Therapeutics, (NASDAQ: CAPR) a clinical-stage biotechnology company at the forefront...
