Content reflects the views of the posting party and is provided for informational purposes only. It does not constitute a solicitation, recommendation, endorsement, or investment advice by BioTech Funding Portal. All content is subject to the portal’s Community Content Guidelines.
Meet Dr. Krista Casazza, PhD — Director of Regulatory Science
Glafabra
Krista most recently served as Scientific Director at the Critical Path Institute, leading biomarker discovery for lysosomal storage disorders for inclusion in FDA trials.
She previously held research leadership roles at Florida Gulf Coast University and the University of Alabama at Birmingham's Department of Pediatrics.
At Glafabra, she leads the regulatory science translating our data into the e...more
What Work Remains Before an IND
Per FDA's INTERACT feedback, remaining work includes finalizing potency assays and release specifications, running a GMP manufacturing campaign under a closed process, completing additional nonclinical characterization FDA requested, and holding a Pre-IND meeting.
None of this is unusual for a pre-IND cell and gene therapy program -- this is the standard pathway, informed directly by regulator fee...more
Why Same-Day Outpatient Conditioning Matters
4 of 5 FACTS trial patients received their conditioning regimen and went home the same day, using a non-myeloablative, melphalan-based approach rather than bone-marrow-transplant-intensity chemotherapy.
For patients, that's materially less treatment burden. For the development program, it's also a differentiator from more intensive HSC-gene-therapy conditioning approaches used elsewhere in the fie...more
What FDA's INTERACT Feedback Means for the CMC Pathway
FDA's written INTERACT feedback (following the July 16 meeting) noted our nonclinical data support a proposed early, exploratory clinical study, recommended plasma lyso-Gb3 as the primary pharmacodynamic readout, endorsed at least two years of eGFR follow-up, and confirmed CMC knowledge (vector backbone, packaging system, process steps) can be leveraged across Glafabra's Fabry, Pompe, and Gaucher ...more
The Clinical Data Dilemma: Balancing Speed and Credibility in Communicating Early-Stage Life Science Clinical Results
Prepared by Putnam Insights LLC
For any early-stage bio- or med-tech company, the arrival of initial positive clinical data presents an immediate dual challenge.
First, there is the fundamental need to establish a highly credible, enduring platform for your science. To gain traction with medical opinion leaders and regulatory bodies, the data must eventually live in a respected, peer-reviewed medi...more
What 'Testing the Waters' Actually Means
Testing the Waters is a formal SEC mechanism under Regulation Crowdfunding that lets a company gauge investor interest before filing a formal offering statement (Form C). No money can be solicited or accepted at this stage, and no offer to buy securities can be made.
That's why every post here carries the full disclosure below, and why registering interest is non-binding -- it's a real, if informa...more
What the Five-Year FACTS Data Does — and Does Not — Show
In the FACTS trial (5 patients, Canada, NCT02800070): a 48% reduction in plasma lyso-Gb3 vs. no-ERT baseline (p<0.0001) at 5-year follow-up; 4 of 5 patients below the lyso-Gb3 threshold at year 5; all 5 maintained durable enzyme expression; zero product-attributable serious adverse events; polyclonal integration with no clonal dominance.
This shows the approach can produce a durable biomarker r...more
What Changed After the First Two Campaign Months
Two months of daily public content. Here's the honest read.
Engagement grew meaningfully once we started introducing the team behind Live-cel and reacting to real industry news, rather than only running scheduled educational content. Our DECODE education series also moved from a manual answer-in-comments format to an integrated Q&A format, based on direct reader feedback.
None of this changes ...more
