Krystal Biotech, Inc. · NASDAQ:KRYS
Krystal Biotech — Technical Presentation (January 2026)
31 slides from Krystal Biotech's technical presentation, January 2026
Slide 1: KB407 for Cystic Fibrosis: CORAL-1 Clinical Data Update
KB407 for Cystic Fibrosis CORAL-1 Clinical Data Update January 2026 © Copyright 2026 Krystal Biotech, Inc. All rights reserved.
Slide 2: Forward Looking Statements and Disclosures
Forward Looking Statements and Disclosures This presentation and our discussion contain forward-looking statements that involve substantial risks and uncertainties. Any statements about future expectations, plans, and prospects for Krystal Biotech, Inc. (the “Company”), including but not limited to
Slide 3: Agenda
Agenda Opening Remarks Krish Krishnan; Chairman and CEO Treatment Gap for Modulator-Ineligible Patients with Cystic Fibrosis Jorge Lascano, MD; Associate Director of Adult Cystic Fibrosis Program, University of Florida KB407 Program Overview Suma Krishnan, MS, MBA; President, Research & Development
Slide 4: Today's Readout Has Transformational Implications for Krystal
Today’s Readout Has Transformational Implications for Krystal Leadership Opportunity in Cystic Fibrosis • KB407 is now the first gene therapy with molecular confirmation of + Lung Platform Validation wild-type CFTR protein expression in the lungs of patients with cystic fibrosis • Lung gene delivery
Slide 5: Cystic Fibrosis Beyond Modulators: Persistent Unmet Need
Cystic Fibrosis Beyond Modulators: Persistent Unmet Need Jorge Lascano, MD Professor of Medicine Associate Director of the Adult Cystic Fibrosis Program Director of the Cystic Fibrosis Therapeutics Development Center University of Florida CORAL-1 Investigator
Slide 6: The Modulator Era: A Major Advance
The Modulator Era: A Major Advance Modulator therapy has revolutionized care for people with CF (pwCF) who have mutations that are eligible for treatment: • Improved pulmonary function (ppFEV1)1 • Decreased pulmonary exacerbations2 • Improved quality-of-life indices1 • Increased life expectancy3 A s
Slide 7: Who Is Left Behind?
Who Is Left Behind? Unresponsive genotype: Mutations which result in no mRNA or functional protein produced (e.g., class 1 mutations) Poor response: ‘Modulator-refractory’ individuals exist due to extensive heterogeneity in CF populations (e.g. non-CFTR 10+% are modulator ineligible, genetic variant
Slide 8: Clinical Burden in the Non-Modulator Population
Clinical Burden in the Non-Modulator Population • Ongoing pulmonary function decline • High exacerbation burden • Continued need for intensive daily therapy • Substantial negative influence on quality-of-life; patients can feel left behind compared to those who benefit from modulator therapy • More
Slide 9: Why Nucleic Acid-Based Therapies Are Urgently Needed
Why Nucleic Acid-Based Therapies Are Urgently Needed Gene replacement therapies offer a mutation-agnostic strategy to target CF, regardless of modulator + + + + + + + status Adapted from Rang, Kotsimbos, and Wilson (2020)
Slide 10: Lessons from Prior Nucleic Acid-Based Efforts
Lessons from Prior Nucleic Acid-Based Efforts Concept Pitfalls faced in previous efforts Idealized quality of future NABT Delivery & - Nebulization through thick and infected airway leads Stable vector (viral or non-viral) capable Stability to poor delivery of expressing CFTR in 5-15% of target - De
Slide 11: Looking Forward
Looking Forward Lessons Learned Unmet * & Next Generation Clinical Need Approaches NABT Historical Pitfalls
Slide 12: Krystal's Redosable HSV-1 Platform for Lung Gene Delivery
Krystal’s Redosable HSV-1 Platform for Lung Gene Delivery Krystal’s Historical Challenges with Inhaled Gene Therapy1 Proprietary ▪ Inhaled gene therapy has been explored for decades, with little success HSV-1 Vectors ▪ Focus to date has been on adenovirus, AAV, and non-viral approaches ▪ Multiple ch
Slide 13: Inhaled Candidate KB407 for Cystic Fibrosis
Inhaled Candidate KB407 for Cystic Fibrosis Preclinical Summary Cellular Tropism: KB407 efficiently transduces human primary airway KB407 ✓ epithelial cells leading to dose dependent CFTR expression Full-Length Payload: CFTR protein expressed in KB407 transduced cells is full- 2 x CFTR genes ✓ lengt
Slide 14: KB407 Phase 1 Study CORAL-1
KB407 Phase 1 Study CORAL-1 109 PFU KB407 Day 0 Study Objectives Cohort 1 KB407 Observation n=3 • Evaluate safety and tolerability of ascending doses of Open Label D0 D2 D7 D14 D21 D28 D58 nebulized KB407, as well as preliminary efficacy evaluation DMC Review • Assessment of KB407 transduction and C
Slide 15: CORAL-1 Cohort 3 Patient Demographics
CORAL-1 Cohort 3 Patient Demographics KB407 109 PFU Days 0, 1, 2, 3 Mandatory bronchoscopy 1-4 days after last dose Safety and Cohort 3 Molecular Four 109 PFU Doses Observation Assessments n = at least 6 D0-D3 D7 D14 D21 D28 D58 Modulator Baseline Modulator Successful Patient ID CFTR Genotype Age Se
Slide 16: Scope of Today's Molecular Data Readout
Scope of Today’s Molecular Data Readout Bronchoscopy Overview • Endoscopic medical procedure to collect biopsies Immunofluorescence Analyses Conducted on Biopsies from conducting airways of the lungs of CF patients • Biopsies were assessed for expression of CFTR or a viral • Biopsies typically colle
Slide 17: CFTR G542X/G542X Control Data
CFF Validated Protocol Used for CFTR Assessments by Immunofluorescence Representative Control Data CFTRWT/WT CFTRG542X/G542X All imaging conducted at 40× magnification; CFTR staining using ab596 Null patient negative control biopsy provided by CFF TDN, positive control biopsy procured commercially C
Slide 18: Clear Evidence of CFTR Delivery and Expression in Lungs of Class I CF Patient
Clear Evidence of CFTR Delivery and Expression in Lungs of Class I CF Patient CFTR expression results for patient 03-01 with CFTR genotype 2184delA/W1282X Patient 03-01 Airway Sampling 42.1% CFTR Positive Cells* + all seven biopsies H&E DAPI CFTR Merge positive for CFTR All imaging conducted at 40×
Slide 19: Clear Evidence of CFTR Delivery and Expression in Lungs of Class I CF Patient
Clear Evidence of CFTR Delivery and Expression in Lungs of Class I CF Patient CFTR expression results for patient 03-01 with CFTR genotype 2184delA/W1282X Patient 03-01 Airway Sampling 42.1% CFTR Positive Cells* + all seven biopsies H&E DAPI CFTR Merge positive for CFTR All imaging conducted at 40×
Slide 20: CFTR Expression in All Relevant Conducting Airway Cell Types
CFTR Expression in All Relevant Conducting Airway Cell Types CFTR expression results for patient 03-01 with CFTR genotype 2184delA/W1282X Ciliated Cells Club Cells Goblet Cells DAPI/CFTR/Acetyl-α-Tubulin DAPI/CFTR/SCGB1A1 DAPI/CFTR/MUC5AC All imaging conducted at 40× magnification Post-dose biopsies
Slide 21: Wild-Type CFTR Expression in Lungs of Second Patient with Class I CF
Wild-Type CFTR Expression in Lungs of Second Patient with Class I CF CFTR expression results for patient 03-02 with CFTR genotype R553X/M1V Patient 03-02 Airway Sampling 29.4% CFTR Positive Cells* H&E DAPI CFTR Merge + all five biopsies positive for CFTR All imaging conducted at 40× magnification Po
Slide 22: Wild-Type CFTR Expression in Lungs of Second Patient with Class I CF
Wild-Type CFTR Expression in Lungs of Second Patient with Class I CF CFTR expression results for patient 03-02 with CFTR genotype R553X/M1V Patient 03-02 Airway Sampling 29.4% CFTR Positive Cells* + all five biopsies H&E DAPI CFTR Merge positive for CFTR All imaging conducted at 40× magnification Po
Slide 23: Confirmed KB407 Transduction by Viral Marker in All Other Patients
Confirmed KB407 Transduction by Viral Marker in All Other Patients CFTR expression results for patient 03-03 with CFTR genotype C1210-12T/1408A>G Patient 03-03 Airway Sampling 36.5% Viral Marker H&E DAPI Viral Marker Merge Positive Cells* + all five biopsies positive for marker All imaging conducted
Slide 24: Confirmed KB407 Transduction by Viral Marker in All Other Patients
Confirmed KB407 Transduction by Viral Marker in All Other Patients CFTR expression results for patient 03-04 with CFTR genotype R334W/R1162X Patient 03-04 Airway Sampling 33.8% Viral Marker H&E DAPI Viral Marker Merge Positive Cells* + all six biopsies positive for marker All imaging conducted at 40
Slide 25: Confirmed KB407 Transduction by Viral Marker in All Other Patients
Confirmed KB407 Transduction by Viral Marker in All Other Patients CFTR expression results for patient 03-05 with CFTR genotype F508del/F508del Patient 03-05 Airway Sampling 36.8% Viral Marker Positive Cells* H&E DAPI Viral Marker Merge + all four biopsies positive for marker All imaging conducted a
Slide 26: Confirmed KB407 Transduction by Viral Marker in All Other Patients
Confirmed KB407 Transduction by Viral Marker in All Other Patients CFTR expression results for patient 03-06 with CFTR genotype F508del/F508del Patient 03-06 Airway Sampling DAPI Viral Marker 31.4% Viral Marker Positive Cells* + all four biopsies positive for marker DAPI Viral Marker All imaging con
Slide 27: KB407 Transduction Confirmed in All Cohort 3 Patients with Bronchoscopies
KB407 Transduction Confirmed in All Cohort 3 Patients with Bronchoscopies Molecular Data Summary Patient Number 03-01 03-02 03-03 03-04 03-05 03-06 2184delA / R553X / C1210-12T / R334W / F508del / F508del / CFTR Variants W1282X M1V 1408A>G R1162X F508del F508del Baseline ppFEV1 64 45 45 69 54 59 Tot
Slide 28: KB407 Continues To Be Well Tolerated in Highest Dose Cohort
KB407 Continues To Be Well Tolerated in Highest Dose Cohort ▪ All but one KB407-related adverse events were mild-to-moderate; all were transient ▪ One serious adverse event of asthma exacerbation reported 24 hours after bronchoscopy ▪ Data monitoring committee considered the adverse event procedure
Slide 29: Next Steps and Accelerating The Path to Potential Registration of KB407
Next Steps and Accelerating The Path to Potential Registration of KB407 Advancing Into Repeat Dosing ▪ Today’s data clearly demonstrate efficient vector delivery and expression of CFTR in patients treated with KB407 ▪ Already working with CFF TDN on repeat dosing CORAL-3 study design which has been
Slide 30: Building Momentum in Krystal's Rare Disease Pipeline
Building Momentum in Krystal’s Rare Disease Pipeline ▪ Confirmation of CFTR delivery sets KB407 on accelerated path towards $2B+ market ▪ Adds to potential registrational readouts for KB803 in ocular DEB and KB801 in NK ▪ Building a portfolio of rare disease medicines that Krystal can launch directl
Slide 31: Developing Genetic Medicines to Treat Diseases with High Unmet Medical Needs
Developing Genetic Medicines to Treat Diseases with High Unmet Medical Needs © Copyright 2026 Krystal Biotech, Inc. All rights reserved.
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