Packaging Therapies Virus 
BioPharma Drive: Drug Pricing
JUNE 11, 2025
The startup, which is backed by the venture arms of Sanofi, Roche and Novartis, is using dual adeno-associated viruses to help overcome the packaging constraints of current genetic medicines.
Broad Institute
JANUARY 14, 2025
Building on previous work by the vector-engineering lab of Ben Deverman at the Broad, the team developed a pair of adeno-associated viruses (AAVs) to package and deliver the base-editing machinery to brain cells. Theres still a long way to go to make this a therapy, Minikel said. But its really exciting to see how much is possible.
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Broad Institute
MAY 27, 2025
If a gene-editing therapy could install an interruption that mimics those that occur naturally in some patients, it might stop the repeat from expanding and halt or slow down disease progression. These individuals are also less likely to pass their disease along to their children than people with uninterrupted repeats.
Codon
JULY 28, 2025
The gene therapy is designed to protect this infant from the neurological disorders that otherwise would have resulted from this rare, and often fatal, condition. And while matching a gene therapy to its targeted mutation is an intricate and nuanced process, much boils down to two core elements: a vector and its cargo.
Codon
JUNE 5, 2025
The treatment, now known as Casgevy, became the first CRISPR-based therapy to gain FDA approval, in 2023. If the same virus invades the cell a second time, the gRNA’s spacer sequence will bind to the matching viral DNA sequence, then be cut by the Cas protein.
Codon
NOVEMBER 1, 2024
They engineered genetically-encoded RNA exporters, based on viruses, that package and secrete RNA molecules in protective nanoparticles, allowing non-destructive monitoring of those RNA molecules in real-time. Today, a single injection of an FDA-approved gene therapy, called Hemgenix , cures this disease.
New Drug Approvals
JULY 18, 2025
It was approved in China in 2021 as a first-in-class treatment for human immunodeficiency virus (HIV). 11] It is believed to work by inhibiting the RNA-dependent RNA polymerase (RdRp) enzyme in the SARS-CoV-2 virus. [12] Detailed numbers are provided by Genuine in the slides and the medication package insert. [14] Swallow, S.;
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