Gene therapy refers to the delivery of genes — typically in the form of nucleic acid polymers — into a patient’s cells, with the goal of treating or preventing disease by correcting, replacing, or supplementing the genetic instructions those cells carry. As one of the foundational modalities within the broader cell and gene therapy field,…
The FDA’s January 2024 guidance on the development of CAR T cell products is 34 pages of non-binding recommendations that nonetheless carry substantial operational weight. For developers navigating the space between IND submission and BLA, the guidance translates the agency’s accumulated experience reviewing six approved autologous CAR-T products into a set of expectations that, while…
Gene editing technologies give researchers and clinicians the ability to make precise, targeted changes directly to an organism’s DNA — adding, removing, or altering specific locations in the genome rather than simply introducing new genetic material alongside the existing sequence, as classic gene therapy does. Three technologies have driven the field’s progress from research tool…
Clinical trials for CAR-T cell therapies are described in terms of patient outcomes: response rates, duration of remission, cytokine release syndrome incidence, progression-free survival. These are the variables that appear in publications, investor presentations, and regulatory submissions. They are the endpoints that determine whether a program succeeds. What they do not capture — what almost…
Messenger RNA, or mRNA, is a family of molecules that carry genetic instructions from a cell’s DNA to its protein-manufacturing machinery, telling the cell which proteins to produce. mRNA therapy harnesses this natural biological process directly, delivering synthetic mRNA into a patient’s cells to instruct those cells to manufacture a specific therapeutic protein — an…
Every genome editing event produces two categories of outcome: the intended modification at the intended genomic location, and everything else. The first category is the therapeutic goal. The second category is the regulatory problem. Understanding why the FDA has made the characterization of both categories a central requirement for gene editing IND submissions — and…
A bacteriophage is a virus that specifically attacks bacteria — found virtually everywhere bacteria exist. While bacteriophage research dates back nearly a century, the field has gained renewed therapeutic relevance in recent years as researchers have found ways to engineer phages for targeted, programmable applications, including a meaningful connection to the gene editing technologies now…
The FDA’s 2024 guidance on human gene therapy products incorporating genome editing addresses two fundamentally different types of programs under a single document. Ex vivo genome editing — in which cells are removed from the patient, modified outside the body, and reinfused — and in vivo genome editing — in which the editing machinery is…
Advanced therapies is the umbrella term encompassing an emerging category of therapeutics that has pushed the broader biopharma ecosystem into genuinely new scientific and regulatory territory — a category that now includes cell therapy, gene therapy, gene editing, mRNA-based therapeutics, and engineered bacteriophage, among other rapidly developing modalities. How Does the Biopharma World Traditionally Categorize…
On December 8, 2023, the FDA approved Casgevy — exagamglogene autotemcel, developed by Vertex Pharmaceuticals and CRISPR Therapeutics — for the treatment of sickle cell disease in patients aged 12 and older with recurrent vaso-occlusive crises. Six weeks later, the FDA approved it again, this time for transfusion-dependent beta-thalassemia. The approvals marked the first time…