Gene Therapy Meets Regulatory Reality: The FDA–uniQure Clash Over a Promising Huntington’s Treatment
The FDA’s dispute with biotech firm uniQure over Huntington’s gene therapy highlights growing tensions between innovation, ...
Successful program buildout prioritizes defined patient flow, infusion access, cold-chain capabilities, and accountability ...
In an interview ahead of Advanced Therapies Week, Anna Catalanotto of Cardinal Health discussed growth in cell and gene ...
As cell and gene therapies move into more hospitals and cancer centers, health system leaders are learning that clinical success is only part of the equation. These treatments may offer life-changing ...
RNA therapeutics target translation rather than DNA, aiming to correct shared protein production errors. By enabling cells to read through premature stop codons, engineered tRNAs could restore ...
Viral vectors dominate gene therapy, with lentivirus, adenovirus, and AAV being key players, each with unique advantages and limitations. Non-viral vectors, such as lipid nanoparticles and GalNAc, ...
Revolutions in gene therapy are rapidly changing the landscape of modern medicine. Revolutions in gene therapy are rapidly changing the landscape of modern medicine, forcing society and science alike ...
NEW ORLEANS, LA—An investigational CRISPR-Cas9 gene-editing therapy that targets angiopoietin-like protein 3 (ANGPTL3), which has a role in regulating lipid metabolism, appears to safely lower levels ...
Clustered regularly interspaced short palindromic repeats (CRISPR) and CRISPR-associated (Cas) proteins are core components of fast-evolving therapeutic gene editing tools. Scientists have used CRISPR ...
Gene editing refers to the precise alteration of an organism's DNA sequence at a specific locus. In cancer treatment, this technology is leveraged to modify the genetic makeup of cancer cells, either ...
Despite the best efforts of modern medicine, Huntington’s disease is a condition that still comes with a tragic prognosis. Primarily an inherited disease, its main symptoms concern degeneration of the ...
Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, will present at the Muscular Dystrophy Association (MDA) Clinical & Scientific Conference, taking ...
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