Alpha-1 antitrypsin deficiency

Brantly Lab


Mark Brantly, MD
Professor of Medicine
Vice Chair of Research, Department of Medicine
Alpha-1 Foundation Research Professor

Personal Statement

I am senior investigator specializing in rare lung disease translational research with a focus on alpha-1-antitrypsin deficiency, gene therapy, pulmonary fibrosis and alveolar macrophage function.

I have mentored a number faculty in bench to bedside research at the NIH Clinical Center and the University of Florida. I am a former K24 grantee, a mentor on 2 T32 programs and the UF MD/PhD program. My role on applicant’s grant is to provide molecular biology support, access to lung tissue and guidance on the transition to patient-oriented research.

REsearch Support


  • Inhibrx 101 LP, ” An Open-Label, Multicenter, Phase 1 Study to Assess the Safety, Pharmacokinetics, and Pharmacodynamics of Single and Multiple Ascending Intravenous Doses of Inhibrx rhAAT-Fc (INBRX-101) in Adults with Alpha-1 Antitrypsin Deficiency (AATD),” Brantly (PI) 1/1/2018-12/31/2019
  • Adverum Biotechnologies, Inc., “Phase 1/2 Study of Intravenous or Intrapleural Administration of a Serotype rh.10 Replication Deficient Adeno-associated Virus Gene Transfer Vector Expressing the Human Alpha-1 Antitrypsin cDNA to Individuals with Alpha-1 Antitrypsin Deficiency,”  Brantly (PI) 1/1/2018-12/31/2019
  • Kamada, LTD. “Follow up, surveillance study on patients who were exposed to inhaled AAT during the Kamada-AAT (inhaled)-006 study,” 1/1/2018-12/31/2018
  • Alpha-1 Foundation Research Professorship/Fernandez Liver Research Grant Research Endowment/4PBA Mediated Alpha-1 Antitrypsin Secretion Rescue Brantly (PI) 12/8/1998-
  • Alpha-1 Foundation / HHS-State of Florida/ Talecris Biotherapeutics/ HICL Principal Investigator Alpha-1 Foundation Tissue Bank/ ACT program/National AAT Screening Program/HICL AAT Screening Program, Brantly (PI) 7/1/2000
  • Grifols National AAT Screening Program,  Brantly (PI) 7/1/2000-12/31/18
  • NIH NCRR/Cincinnati Children’s Hospital Medical Center/LAM Foundation, “Quantitative Chest Computed Tomography Unmasking Emphysema Progression in Alpha-1 Antitrypsin Deficiency” Brantly (PI) 11/1/07-6/30/17


  • NIH NCRR/Cincinnati Children’s Hospital Medical Center/LAM Foundation “The Multicenter International Lymphangioleiomyomatosis Efficacy of Sirolimus (MILES),” Brantly (Site PI) 11/01/07- 06/30/2010
  • Kamada, LTD.,”Phase II/III Randomized Double-Blind Comparison of Alpha-1 Proteinase Inhibitor(Kamada-API) with Prolastin,”Brantly (PI) 04/23/07- 01/17/10
  • NIH/NIHLBI, “The Anti-Inflammatory Role of Alpha-1 Antitrypsin in the Lung (K24-HL004456)” Brantly (PI) 3/30/01-07/21/07
  • Florida Department of Health, “Tobacco Smoke-Induced Cell Injury in Lung Compartments,” Brantly (CO-I) 08/01/04-03/31/08
  • Washington University, “Washington University Testing Services Agreement,”Brantly (PI), 02/12/08 -06/30/10

Publication Highlights

  • Flotte, T.R., Trapnell, B.C., Humphries, M., Carey, B., Calcedo, R., Rouhani, F., Campbell-Thompson, M., Yachnis, A.T., Brantly, M.L., Sandhaus, R.A., McElvaney, N.G., et al. 2011. Phase 2 Clinical Trial of a Recombinant Adeno-Associated Viral Vector Expressing alpha(1)-Antitrypsin: Interim Results. Human gene therapy.
  • McCormack, F.X., Inoue, Y., Moss, J., Singer, L.G., Strange, C., Nakata, K., Barker, A.F., Chapman, J.T., Brantly, M.L., Stocks, J.M., et al. 2011. Efficacy and safety of sirolimus in lymphangioleiomyomatosis. The New England journal of medicine 364:1595-1606.
  • Grimstein, C., Choi, Y.K., Wasserfall, C.H., Satoh, M., Atkinson, M.A., Brantly, M.L., Campbell-Thompson, M., and Song, S. 2011. Alpha-1 antitrypsin protein and gene therapies decrease autoimmunity and delay arthritis development in mouse model. Journal of translational medicine 9:21.
  • Stocks, J.M., Brantly, M.L., Wang-Smith, L., Campos, M.A., Chapman, K.R., Kueppers, F., Sandhaus, R.A., Strange, C., and Turino, G. 2010. Pharmacokinetic comparability of Prolastin(R)-C to Prolastin(R) in alpha-antitrypsin deficiency: a randomized study. BMC clinical pharmacology 10:13.
  • Brantly, M.L., Mahr, A.D., Edberg, J.C., Stone, J.H., Hoffman, G.S., St Clair, E.W., Specks, U., Dellaripa, P.F., Seo, P., Spiera, R.F., Rouhani, F.N., et al. 2010. Alpha-antitrypsin deficiency-related alleles Z and S and the risk of Wegener’s granulomatosis. Arthritis and rheumatism 62:3760-3767.
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