Jorge Lascano

CFF Center 26-27

A PHASE 1/2 DOSE-EXPLORATION AND DOSE-EXPANSION STUDY TO EVALUATE THE SAFETY AND EFFICACY OF BEAM 302 IN ADULT PATIENTS WITH ALPHA-1 ANTITRYPSIN DEFICIENCY(AATD) ASSOCIATED LUNG DISEASE AND/OR LIVER DISEASE

A Phase 2, Single-Arm, Open-Label Extension Study, Evaluating the Long-Term Safety and Clinical Efficacy of INBRX-101 in Adults with Alpha-1 Antitrypsin Deficiency (AATD) Emphysema

A Prospective, Observational, Long-Term Follow-Up (LTFU) Study to Evaluate the LongTerm Safety of the Krystal Biotech, Inc. Gene Therapy Backbone using HSV

CTOA

Impact of a Multicenter Best Practice Alert to Improve AATD Testing and Detection

CFF Center 25-26 - Trillo

A Phase 1 Study of Inhaled KB408 for the Treatment of Alpha-1 Antitrypsin Deficiency”

A Research Study to Advance the CF Therapeutics Pipeline for People without Modulators

A Phase 2, Single Arm, Open Label Extension Study, Evaluating the Long-term Safety and Clinical Efficacy of INBRX-101 in Adults with Alpha-1 Antitrypsin Deficiency (AATD) Emphysema CLINICALA Phase 2, Single Arm, Open Label Extension Study, Evaluating 101-01-202

Therapeutics Development Center Award 2024-2025

CFF Center 24-25 - Trillo

Nicotine as a Cause of Airway Inflammation and Lung Emphysema in a Novel AATD Mouse Model

A Phase 1 Study of Inhaled KB407, a Replicati on-Defecti ve, Non-IntegCysti c Fibrosis Transmembrane Conductance Regulator, for the Treatment of Cystic Fibrosis

CFF Center 23-24 - Trillo

A Phase 2, Double-Blind, Randomized, Active-Control, Parallel Group Study to Assess the Pharmacokinetics, Pharmacodynamics, Immunogenicity, and Safety of INBRX-101 Compared to Plasma Derived Alpha1-Proteinase Inhibitor (A1PI) Augmentation Therapy in Adults with Alpha-1 Antitrypsin Deficiency (AATD) Emphysema

Therapeutics Development Center Award

Personalized Mobile Health Platform to Promote Physical Activity in Adolescents and Young Adults with Cystic Fibrosis

A Phase 1b/2a, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate Nebulized Bacteriophage Treatment in Outpatient Adult Cystic Fibrosis (CF) Subjects with Chronic Pseudomonas aeruginosa (PsA) Pulmonary Infection

CFF Center 22-23 - Trillo

A Phase 2a, Single-Blind, Placebo-Controlled, Parallel-Group Study to Assess Safety, Tolerability, and Pharmacokinetics of Brensocatib Tablets in Adults With Cystic Fibrosis

ABATE: A Phase 1b, Multi-center Study of IV Gallium Nitrate in Patients with Cystic Fibrosis who are colonized with Nontuberculous Mycobacteria (The Abate Study)

A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Assess the Efficacy, Safety, and Tolerability of Brensocatib Administered Once Daily for 52 Weeks in Subjects with Non-Cystic Fibrosis Bronchiectasis – The ASPEN Study

A Multi-center, Single-Dose and Repeat-Dose Over Eight Weeks, Sequential Cohort Study to Evaluate Safety and Tolerability as well as Pharmacokinetics of Two Different Doses of Alpha1-Proteinase Inhibitor Subcutaneous (Human) 15% Administered Subcutaneously in Subjects with Alpha1-Antitrypsin Deficiency

A Phase 1/2a randomized, double-blind, two-part, dose-ascending, multicenter study of the safety and pharmacokinetics of AR-501 (gallium citrate), administered via inhalation, in healthy adult and P. aeruginosa infected cystic fibrosis subjects