title: Critical considerations for developing nucleic acid macromolecule based drug products.
abstract: Protein expression therapy using nucleic acid macromolecules (NAMs) as a new paradigm in medicine has recently gained immense therapeutic potential. With the advancement of nonviral delivery it has been possible to target NAMs against cancer, immunodeficiency and infectious diseases. Owing to the complex and fragile structure of NAMs, however, development of a suitable, stable formulation for a reasonable product shelf-life and efficacious delivery is indeed challenging to achieve. This review provides a synopsis of challenges in the formulation and stability of DNA/m-RNA based medicines and probable mitigation strategies including a brief summary of delivery options to the target cells. Nucleic acid based drugs at various stages of ongoing clinical trials are compiled.
authors.affiliation: Formulation Sciences, Biopharmaceutical Development, Medimmune(4), One Medimmune Way, Gaithersburg, MD 20878, USA. Electronic address: muralidharab@medimune.com.
authors.forename: Bilikallahalli K
authors.initials: BK
authors.lastname: Muralidhara
authors.affiliation: Formulation Sciences, Biopharmaceutical Development, Medimmune(4), One Medimmune Way, Gaithersburg, MD 20878, USA.
authors.forename: Rinku
authors.initials: R
authors.lastname: Baid
authors.affiliation: Formulation Sciences, Biopharmaceutical Development, Medimmune(4), One Medimmune Way, Gaithersburg, MD 20878, USA.
authors.forename: Steve M
authors.initials: SM
authors.lastname: Bishop
authors.affiliation: Biotherapeutics Pharmaceutical Sciences, Pfizer, 1 Burtt Road, Andover, MA 01810, USA.
authors.forename: Min
authors.initials: M
authors.lastname: Huang
authors.affiliation: Biotherapeutics Pharmaceutical Sciences, Pfizer, 700 Chesterfield Parkway West, Chesterfield, MO 63017, USA.
authors.forename: Wei
authors.initials: W
authors.lastname: Wang
authors.affiliation: Biotherapeutics Pharmaceutical Sciences, Pfizer, 700 Chesterfield Parkway West, Chesterfield, MO 63017, USA.
authors.forename: Sandeep
authors.initials: S
authors.lastname: Nema
mesh_terms: D004339:Drug Compounding; D004355:Drug Stability; D009696:Nucleic Acids / Q000737:chemistry*
chemical_list: D009696:Nucleic Acids