Designed Experiments- A Vital Role in Validation

FDA draft guidance on individualized genome editing lets BLAs add patient-specific variants without new trials, shifting evidence to CMC assay data.

Subathra Ramamoorthy, Veranova, on when to escalate to structural elucidation and pair orthogonal techniques when characterizing an impurity.

Manish Baviskar, Hubot CDMO, on ALCOA++ data integrity, model version change control, continuous monitoring, and human sign-off.

Manish Baviskar, Hubot CDMO, explains which AI skills validation engineers need first and why AI model updates must go through GMP change control.

Vibha Jawa, PhD, chief scientific officer at EpiVax, explains why the need for animal studies varies by modality and what it takes for organ-on-chip and other human-relevant models to replace these studies in IND-enabling toxicology work.

Cole and Jaenisch dig into AI poisoning in pharma manufacturing, how it hides in plain sight, and who should be accountable for catching it.

FDA grants ravulizumab accelerated approval in IgA nephropathy after a 43.4% proteinuria cut, signaling new IV biologic demand for drug manufacturers.

FDA approves GSK's bepirovirsen for chronic hepatitis B after a 19% functional cure rate, raising oligonucleotide scale-up stakes for manufacturers.