Investigational New Drug Applications (INDs) for Gene Therapy
Products, 2002-2017
Chart1
CY2002
CY2003
CY2004
CY2005
CY2006
CY2007
CY2008
CY2009
CY2010
CY2011
CY2012
CY2013
CY2014
CY2015
CY2016
CY2017
22
41
39
34
36
44
50
44
44
44
53
53
72
80
79
106
All submitted
FOLDER_IDIRA_TYPECENTER_RECEIPT_DTCURRENT_IRA_STATUSCURRENT_IRA_STATUS_DTPAST_STATUSPAST_STATUS_DTCURRENT
OFFICEPARENT
OFFICESPONSORIRA_TITLEPRODUCTIRA_PROPOSED_USESPONSOR_TYPEEMERGENCY_USEGENE_THERAPYCLINICAL_PHASESOM_CELL_THERAPYFAST_TRACKBREAKTHROUGHCOMBINATIONORPHAN_DESIGNATIONRARE_PEDIATRICRARE_DISEASEUSER_FEERRS
17437IND6-Apr-17ACTIVE4/6/17OTATOCTGTMemorial Sloan-Kettering
Cancer CenterHuman Wilms Tumor Protein 1 Peptides (4 Synthetic
Peptides, American Peptide Company) Mixed with Montanide ISA-51 VG
Adjuvant (Seppic); and Administered with GM-CSF (Berlex)Multiple
products listed for IND 17437Emergency - Treatment of multiple
myeloma in a single patientRHEMNUFPCELL AND GENE THERAPY
17354IND9-Feb-17ACTIVE2/9/17OTATOCTGTMemorial Sloan-Kettering
Cancer CenterAllogeneic Epstein-Barr Virus Cytotoxic
T-LymphocytesMultiple products listed for IND 17354Emergency -
treatment of EBV post-transplant lymphoproliferative
diseaseRHEMS3NUFPCELL AND GENE THERAPY
17544IDE9-Jun-17APPROVED7/3/17OTATOCTGTMemorial Sloan-Kettering
Cancer CenterMagnetic-Activated Cell Sorter (CliniMACS, Miltenyi)
TCR alpha/beta and CD19 T-cell depletion PBSC; conditioningMultiple
products listed for IND 17544Leukocyte Adhesion Deficiency Type
1RH2NUFMDEVICES
17756IND4-Oct-17ACTIVE10/10/17OTATOCTGTMemorial Sloan-Kettering
Cancer CenterEmergency Single Patient Use of Vigil Vaccine:
Autologous Tumor Cells Transfected with bifunctional short hairpin
ribonucleic acid furin and granulocyte macrophage-colony
stimulating factor, radiated administered by injection.Tumor Cells
(Autologous) Transfected with Recombinant Human Granulocyte
Macrophage Colony Stimulating Factor Gene (DNA Plasmid,
FANG)Treatment of Ewing sarcomaRHSPG6NUFPCELL AND GENE THERAPY
17770IDE13-Oct-17DISAPPROVED11/9/17OTATOCTGTMemorial
Sloan-Kettering Cancer CenterAllogeneic, HLA-Compatible Unrelated
and HLA Mismatched Related, G-CSF Mobilized, T-cell depleted
Peripheral Blood Stem Cells, CD34+ Selected with the
Magnetic-Activated Cell Sorter (CliniMACS, Miltenyi),
ChemotherapyMultiple products listed for IND 17770Treatment of
patients with Fanconi AnemiaLacking a Genotypically Identical Donor
and severe hematologic disease.RH2S1NUFMDEVICES
17420IND29-Mar-17ACTIVE3/29/17OTATOCTGTMemorial Sloan-Kettering
Cancer CenterRapidly Generated Peptide Stimulated
Multivirus-directed Specific Cytotoxic T-Lymphocytes derived from
Allogeneic PBMCs and Pulsed with CytokinesNo product listed for IND
17420Emergency - Treatment of BK viremia and adenoviremia in a
single patient.RHEMS3NUFPCELL AND GENE THERAPY
17355IND10-Feb-17ACTIVE2/10/17OTATOBRRMemorial Sloan-Kettering
Cancer CenterCytomegalovirsu Cytotoxic T-Lymphocytes
(CMV-CTLs)allogeneic CMV Specific Cytotoxic T lymphocytesEmergency
- treatment of CMV resistant reactivation in drug resistent patient
(donor derived) post-stem cell transplantRHEMS5NUFPCELL AND GENE
THERAPY
17651IND8-Aug-17ACTIVE9/7/17OTATOCTGTMemorial Sloan-Kettering
Cancer CenterAllogeneic Matched Related TCR alpha beta and CD19
B-Cell depleteion of PBSC using the Magnetic-Acrtivated Cell Sorter
(CliniMACS)Multiple products listed for IND 17651Treatment of
Wiskott-Aldrich SyndromeRHEMS1NUFPCELL AND GENE THERAPY
17334IND27-Jan-17ACTIVE2/24/17OTATOCTGTMemorial Sloan-Kettering
Cancer CenterEGFRr/BCMA-41BBz CAR T Cells;Dynabeads CD3/CD28 CTS;
Defined Composition CD4 and CD8 T CellsNo product listed for IND
17334Treatment of multiple myelomaRHG42NUFPCELL AND GENE
THERAPY
17346IND22-Feb-17ACTIVE5/16/17HOLD3/24/17OTATOCTGTNational
Cancer InstituteCDX-1401 (Celldex); Poly-ICLC ; SGI-110
(Decitabine), and Atezolizumab (MPDL3280A)No product listed for IND
17346metastatic or unresectable malignancy for which standard
curative or palliative measures do not exist or are no longer
effectiveCM2NUFPCELL AND GENE THERAPY
17311IND19-Jan-17ACTIVE2/18/17OTATOCTGTNational Cancer
InstituteHerpes Simplex Type-1 Virus Encoded with Human
Granulocyte-Macrophage Colony-Stimulating Factor (rHSV-1hGM-CSF);
Oncolytic Vaccine (Imlygic) [Talimogene laherparepvec] in
combination with RadiationHerpes Simplex Viruse Type-1 Virus
Encoded with Human GM-CSF(rHSV-1hGM-CSF)Treatment for localized
soft tissue sarcomaRHG52NUFPCELL AND GENE THERAPY
17889IND19-Dec-17ACTIVE12/19/17OTATOCTGTBaylor College of
MedicinePartially HLA-Matched AdV-Directed VSTs (Viralym-A)No
product listed for IND 17889Emergency - treatment of adenovirus
infectionRHEMS3NUFPCELL AND GENE THERAPY
17774IND18-Oct-17WITHDRAWN11/22/17OTATOCTGTNational Institute of
Allergy and Infectious DiseasesHaploidentical Transplant Selected
with CLiniMACS CD34 Reagent System with Alemtuzumab, Busulfan and
Total Body Irradiation (TBI) with Post-Transplant Cyclophosphamide
(HaploTransplant CGD with post CTX)No product listed for IND
17774Treatment of Chronic Granulomatous DiseaseCM1S1NUFPCELL AND
GENE THERAPY
17374IDE22-Feb-17APPROVED5/16/17OTATOCTGTGrupp MD PhD, Stephan
AMagnetic- Activated Cell Sorter (CliniMACS, Miltenyi) for
TCR??+/CD19+ Depeletion of G-CSF or GM-CSF Mobilized Allogeneic
Unrelated or Partially Matched Related Peripheral Stem Cells;
ChemotherapyMultiple products listed for IND 17374Expanded access
for patients who lack a fully HLA matched sibling donor, and who
are candiates for allogeneic hematopoietic stem cell transplant.
(malignant and non-malignant diseases)RH1S1NUFMDEVICES
17351IND8-Feb-17ACTIVE3/10/17OTATOCTGTNational Institute of
Allergy and Infectious DiseasesAutologous Ex-Vivo Expanded
CD4+CD127lo/-CD25+ Polyclonal Regulatory T Cells (Poly Tregs)T
regulatory cellsTreatment of Adults with Active PemphigusRH1S3NRARE
DISEASEUFPCELL AND GENE THERAPY
17846IND20-Nov-17ACTIVE12/20/17OTATOCTGTNational Institute of
Allergy and Infectious DiseasesAllogeneic Bone Marrow-Derived
Mesenchymal Stem Cells (hBM-MSC)Allogeneic Bone Marrow Derived
Mesenchymal Stem CellsTreatment of adult recipients of living donor
kidney transplant with co-stimulatory blockadeRH1S3NUFPCELL AND
GENE THERAPY
17497IND16-May-17ACTIVE6/14/17OTATOCTGTNational Institute of
Allergy and Infectious DiseasesAllogeneic Bilateral Orthotopic Lung
Transplant (BOLT) in tandem with Partially HLA Matched, CD3+/CD19+
Depleted Cadaveric Bone Marrow Transplant using the
Magnetic-Activated Cell Sorter (CliniMACS, Miltenyi), Hydroxyurea,
Rituximab, Alemtuzumab, ATG, and ThiotepaMultiple products listed
for IND 17497Treatment of patients with lung or intestinal end
organ failure in concert with severe immune deficiencyRH2S1NRARE
DISEASEUFPCELL AND GENE THERAPY
17803IND31-Oct-17ACTIVE11/30/17OTATOCTGTRoswell Park Cancer
InstituteCDX-1401/Poly-ICLC in combination with Decitabine and
NivolumabMultiple products listed for IND 17803Myelodysplastic
Syndromes (MDS) or Low Blast Count AMLRH16UFPCELL AND GENE
THERAPY
17590IND6-Jul-17ACTIVE8/4/17OTATOCTGTRoswell Park Cancer
InstituteNY-ESO-1 Protein with MIS416 Vaccine in Combination with
SirolimusNY-ESO-1 Cancer VaccineInducing Anti-Tumor Immunity in
Ovarian, Fallopian Tube and Primary Peritoneal CancerRH1NUFPCELL
AND GENE THERAPY
17410IND20-Mar-17ACTIVE4/19/17OTATOCTGTRoswell Park Cancer
InstituteNY-ESO-1 TCR/ dn TGF(beta)RII transgenic T cellsMultiple
products listed for IND 17410Advanced malignanciesRHG41NUFPCELL AND
GENE THERAPY
17527IND5-Jun-17ACTIVE7/5/17OTATOCTGTRoswell Park Cancer
InstituteAutologous PBMNC Transduced with Retroviral Vector
NY-ESO-1 TCR in Combination with DecitabineMultiple products listed
for IND 17527Treatment of recurrent or refractory ovarian
cancerRHG41NUFPCELL AND GENE THERAPY
17521IND17-Nov-17HOLD12/13/17HOLD12/13/17OTATOCTGTMaxcyte
IncAutologous Peripheral Blood Leukocytes, Transfected via
Electroporation to Express Human Mesothelin Chimeric Antigen
Receptor mRNAMCY-M11Platinum Resistant High Grade Serous
Adenocarcinoma of the Ovary, Primary Peritoneum, or Fallopian Tube
or Peritoneal MesotheliomaCMG11NUFPCELL AND GENE THERAPY
17891IND21-Dec-17PENDING12/21/17OTATOCTGTRibas MD PhD,
AntoniRV-NYESO TCR PBMC, LV-NYESO TCR/sr39TK PBSCNo product listed
for IND 17891NY-ESO-1-positive relapsed or relapsed and refractory
multiple myelomaRHG41NUFPCELL AND GENE THERAPY
17471IND1-May-17ACTIVE5/26/17OTATOCTGTRibas MD PhD,
AntoniRV-NYESO TCR PBMC; LV-NYESO TCR/sr39TK PBSCNo product listed
for IND 17471Locally advanced or metastatic NY-ES0 -1 positive
cancerRHG41NUFPCELL AND GENE THERAPY
17743IND27-Sep-17HOLD10/27/17HOLD10/27/17OTATOCTGTTissueTech
IncAllogeneic Human Umbilical Cord Placental Tissue, Cryopreserved;
Generic: Cryopreserved Human Umbilical Cord; Code: TTAX02Umbilical
Cord Tissue, Allogeneic, HumanTo promote wound healing and closure
of developmental defects during in-utero repair of severe spina
bifidaCM3S1NRARE DISEASEUFPCELL AND GENE THERAPY
17890IND19-Dec-17PENDING12/19/17OTATOCTGTCity of Hope National
Medical CenterHER2(EQ)BB /CD19t+ T cellsNo product listed for IND
17890Recurrent or refractory malignat gliomaRHG41NUFPCELL AND GENE
THERAPY
17432IND5-Apr-17ACTIVE4/7/17OTATOCTGTCity of Hope National
Medical CenterHLA-matched BK specific Cytotoxic T lymphocytes (BK
CTLs)No product listed for IND 17432Individual patient IND under
expanded access for non-emergency use using BK specific CL lines
generated by ex-vivo expansion in a post-transplant patient (DF)
with BK virus infection.RHEMS3NUFPCELL AND GENE THERAPY
17638IND1-Aug-17ACTIVE8/31/17OTATOCTGTCity of Hope National
Medical CenterAllogeneic CD4+ T-cell-depleted HPC with
Cyclophosphamide, Pentostatin, Anti-thymocyte globulinAllogeneic
Hemopoietic Stem CellsSickle Cell DiseaseRH1S3NUFPCELL AND GENE
THERAPY
17501IND9-Jun-17ACTIVE7/7/17OTATOCTGTCelgeneAutologous CD4+/CD8+
T Cells Expressing CD19 Chimeric Antigen Receptor(JCAR017)No
product listed for IND 17501Relapsed/refractory aggressive B-cell
non-Hodgkin lymphoma (NHL)CMG41NORPHAN DESIGNATIONUFPCELL AND GENE
THERAPY
17761IND5-Oct-17ACTIVE11/3/17OTATOCTGTUniv of
TexasOff-the-shelf, Expanded, Most Closely HLA Matched, Allogenic
Adenovirus-Specific T cellsNo product listed for IND
17761Adenovirus related disease in immunocompromised
patientsRH1S3NUFPCELL AND GENE THERAPY
17876IND12-Dec-17PENDING12/12/17OTATOCTGTUniv of
TexasPembrolizumab, Clostridium novyi-NTNo product listed for IND
17876Refractory Solid TumorsRHG818UFPCELL AND GENE THERAPY
17742IND10-Oct-17ACTIVE11/9/17OTATOCTGTUniv of TexasHPV-DP-5P
and HPV-DP-7P peptide vaccine and Utomilumab VaccineMultiple
products listed for IND 17742Immunotherapy for HPV-16-positive
incurable oropharyngeal cancerRH2NUFPCELL AND GENE THERAPY
17344IND6-Feb-17ACTIVE8/16/17HOLD3/8/17OTATOCTGTUniv of
TexasAutologous IL-21-Primed CD8+ Tumor Antigen-Specific T
CellsMultiple products listed for IND 17344Treatment of Platinum
Resistant Ovarian CancerRH1S1NUFPCELL AND GENE THERAPY
17757IND4-Oct-17ACTIVE11/3/17OTATOCTGTUniv of TexasAllogeneic
Umbilical Cord Blood (UCB) Derived NK Cells Expanded Ex
VivoUmbilical Cord Blood (UCB) Derived NK CellsTreatment of Solid
TumorsRH1S3NUFPCELL AND GENE THERAPY
17656IND11-Aug-17ACTIVE9/10/17OTATOCTGTUniv of TexasAllogeneic
Bone Marrow Derived Multipotent Mesenchymal Stromal Cells
administered by Intravenous Infusion or Transendocardial
InjectionMultiple products listed for IND 17656Patients with Recent
Onset of Anthracycline-Associated CardiomyopathyRH1S3NUFPCELL AND
GENE THERAPY
17621IND21-Jul-17WITHDRAWN8/9/17OTATOCTGTUniv of TexasUtomilumab
and ISA101b Peptide VaccinationMultiple products listed for IND
17621Treatment for HPV-16-Positive Incurable Solid
TumorsRH2NUFPCELL AND GENE THERAPY
17428IND31-Mar-17ACTIVE4/28/17OTATOCTGTUniv of TexasAutologous
T-cells Transduced with Lentiviral Vector Expressing CD33-Specific
Chimeric Antigen Receptor Gene (CD33-CAR-T cells)CD3+ T cells
genetically modified using a lentiviral vector (INXN-2006) to
co-express a CD33-specific CAR and the HER1t depletion
markerPatients with Relapsed or Refractory CD33-Positive Acute
Myeloid LeukemiaRHG41NUFPCELL AND GENE THERAPY
17356IND13-Feb-17ACTIVE3/15/17OTATOCTGTUniv of TexasAutologous
CD8+ Antigen-Specific T Cells and Anti-CTLA4Multiple products
listed for IND 17356For patients with metastatic uveal
melanomaRH1S1NUFPCELL AND GENE THERAPY
17321IND13-Jan-17ACTIVE4/11/17HOLD2/10/17OTATOCTGTUniv of
TexasAllogeneic Donor Umbilical and Cord Blood derived
CAR-CD19-CD28-Zeta-2a-iCasp9-IL15 Transduced NK CellsAllogeneic
Donor Umbilical and Cord Blood derived
CAR-CD19-CD28-Zeta-2a-iCasp9-IL15 Transduced NK CellsTreatment for
relapsed/refractory B-lymphoid malignanciesRHG42NUFPCELL AND GENE
THERAPY
17378IND23-Feb-17ACTIVE3/25/17OTATOCTGTUniv of TexasEx-Vivo
Expanded using MSC Derived from Bone Marrow, Fucosylated and
Unmanipulated Alllogeneic unrelated Cord Blood,
chemotherapyMultiple products listed for IND 17378Enhance homing
and engraftmentRH2S39UFPCELL AND GENE THERAPY
17314IND6-Jan-17ACTIVE5/5/17HOLD2/3/17OTATOCTGTUniv of
TexasNatural Killer (NK) Cells derived from ex vivo-expanded Cord
Blood, Combined with Rituximab-High Dose Chemotherapy and
Autologous Stem Cell TransplantMultiple products listed for IND
17314Immunotherapy for B-cell non-Hodgkin's lymphomaRH2S3NUFPCELL
AND GENE THERAPY
17731IND19-Dec-17PENDING12/19/17OTATOCTGTAmgen IncAutologous,
CAR-T cells targeting delta-like ligand 3 (DLL3), transduced with
third-generation SIN HIV-1 lentiviral vector, AMG 119Multiple
products listed for IND 17731Relapsed/Refractory Small Cell Lung
CancerCMG41NUFPCELL AND GENE THERAPY
17383IND27-Feb-17ACTIVE3/29/17OTATOCTGTFred Hutchinson Cancer
Research CenterAutologous T-cells Transduced with Lentiviral Vector
Expressing CD20-specific Chimeric Antigen Receptor GeneNo product
listed for IND 17383Treatment of Relapsed or Refractory B Cell
Non-Hodgkin LymphomasRHG12NUFPCELL AND GENE THERAPY
17663IND14-Aug-17ACTIVE8/16/17OTATOCTGTFred Hutchinson Cancer
Research CenterAutologous Peripheral Blood T cells Transduced with
Retroviral Vector (BPZ-1002; SFG.iCasp9.2A.SLL-TCR) Expressing
human T Cell Receptor (TCR) alpha and beta chains for the
Preferentially Expressed Antigen in Melanoma (PRAME)-derived
peptide SLLQLIGL (BPX-701 T cells); Following Chemotherapy; AP1903
Administered Only for Uncontrolled BPX-701 ToxicityMultiple
products listed for IND 17663Single patient treatment of relapsed
or refractory acute myeloid leukemiaRHSPG46UFPCELL AND GENE
THERAPY
17855IND22-Nov-17ACTIVE12/21/17OTATOBRRNational Institute of
Diabetes, Digestive, & Kidney DiseasesHepatitis B
Immunoglobulin (human) and Peginterferon alfa-2aMultiple products
listed for IND 17855Treatment of Chronic Hepatitis B
VirusRH2NUFPHEMATOLOGICS
17455IDE20-Apr-17APPROVED5/17/17OTATOCTGTMayo ClinicCytori
Celution SystemNo product listed for IND 17455Bilateral
Pre-collapse Femoral Head OsteonecrosisCM1S1NUFMDEVICES
17473IND23-Jun-17HOLD7/20/17HOLD7/20/17OTATOCTGTPfizer
IncPF-06755347 (Recombinant Multimerized Human IgG1 Fc)recombinant
human Fc fragment containing human (h) Fc1 (hinge, CH2 and CH3
regions)Treatment of chronic inflammatory demyelinating
polyneuropathy (CIDP)CM1NORPHAN DESIGNATIONUFPCELL AND GENE
THERAPY
17659IND1-Sep-17ACTIVE9/29/17OTATOCTGTBioMarin Pharmaceutical
IncAdeno-Associated Virus Serotype 5 (AAV5) Expressing SQ form of
Human Factor VIII (hFVIII-SQ) (BMN 270)Adeno-Associated Virus
Serotype 5 (AAV5) Vector Expressing hFVIII-SQ.treatment of
Hemophilia ACMG53Breakthrough Therapy DesignationNORPHAN
DESIGNATIONUFPCELL AND GENE THERAPY
17217IND9-Jan-17ACTIVE2/8/17OTATOCTGTLudwig Institute for Cancer
Researchdurvalumab (MEDI4736), Adenovirus Vector (Ad5/3-D24-GMCSF),
Expressing GM-CSF (ONCOS-102)Adenovirus Vector Type 3
(Ad5/3-D24-GMCSF)Treatment of peritoneal cancerRHG51NUFPCELL AND
GENE THERAPY
17480IND25-May-17ACTIVE6/23/17OTATOCTGTLudwig Institute for
Cancer ResearchmRNAs Encoding Tumor Specific Antigens (MUC-1,
Survivin, NY-ESO-1, 5T4, MAGE-C2 and MAGE-C1) with Durvalumab and
Tremelimumab Administrated IntradermallyMultiple products listed
for IND 17480Non-small cell lung cancerRHG615UFPCELL AND GENE
THERAPY
17557IND19-Jun-17ACTIVE7/19/17OTATOCTGTGenentech IncRO7198457,
Personalized Cancer Vaccine (PCV)ROT 198457 RNASolid
TumorsCM18UFPCELL AND GENE THERAPY
17624IND24-Jul-17ACTIVE8/23/17OTATOCTGTUniv of Pennsylvania
Medical CenterAutologous Dendritic Cells Derived from Peripheral
Blood Mononuclear Cells, Expanded with Interleukin-4,
Granulocyte-Macrophage Colony-Stimulating Factor; Pulsed with
Synthetic Tumor-Stem Cell Associated Antigens [AIM-2, MAGE-1,
gp100, HER-2, IL-13Ra2] ICT 107-301Dendritic Cells Derived from
Peripheral Blood Mononuclear CellsSingle Patient Use for Newly
Diagnosed Glioblastoma MultiformeRHSPS4NUFPCELL AND GENE
THERAPY
17849IND21-Nov-17ACTIVE12/21/17OTATOCTGTUniv of Pennsylvania
Medical CenterAutologous T cells Transduced with Lentiviral Vector
(AntiBCMAbbz human) Expressing B-cell Maturation Antigen
(BCMA)-specific Chimeric Antigen Receptor (CART-BCMA) with or
without Autologous CD3+ T Cells Cultured with Anti-CD3 and
Anti-CD28 Coated Magnetic Beads; Transduced with self-inactivating
(SIN) Lentiviral Vector (CD19bbz-human Lot# 060713S12) Expressing
Humanized CD19 Chimeric Antigen Receptor (huCART19); and
chemotherapyMultiple products listed for IND 17849Treatment of
multiple myelomaRHG41NUFPCELL AND GENE THERAPY
17871IND6-Dec-17PENDING12/6/17OTATOCTGTFuchs MD, Ephraim
JAllogeneic HLA-matched or HLA-haploidentlcal Bone Marrow
Transplant and Unstimulated Lukapheresed Peripheral Blood (DLI; T
Cells) Donors that have been vaccinated with VGX-3100 (IND 13683;
Inovio vaccine) via electroporatlon by the Cellectra 2000;
ChemotherapyNo product listed for IND 17871Treatment of recurrent,
human papillomavlrus associated (HPV*) oropharyngeal squamous cell
carcinoma (OPSCC).RHG61S1NUFPCELL AND GENE THERAPY
17906IND28-Dec-17ACTIVE12/28/17OTATOBRRNot EnteredBPX501
(Bellicum)No product listed for IND 17906Emergency - Second dose to
boost antileukemia effect, has residual diseaseEM
17893IND23-Dec-17ACTIVE12/23/17OTATOCTGTNot
EnteredtisagenlecleucelMultiple products listed for IND
17893Emergency - Yes, for the treatment of relapsed refractory
ALLEM
17901IND26-Dec-17ACTIVE12/26/17OTATOCTGTNot
Enteredtisagenlecleucel (antiCD19 CAAR T cell)Multiple products
listed for IND 17901Emergency - need approval to porceed with
manufacturingEM
17867IND1-Dec-17ACTIVE12/1/17OTATOCTGTNot EnteredCV-MG01No
product listed for IND 17867Emergency - Treatment of myasthemia
gravisEM
17865IND29-Nov-17ACTIVE11/29/17OTATOCTGTNot EnteredAutologous
Tumor Infiltrating Lymphocytes Cultured with OKT3, Interleukin-2,
and Allogeneic Mononuclear Feeder CellsNo product listed for IND
17865Emergency - Treatment of melanoma in a single
patientEMS3UFPCELL AND GENE THERAPY
17492IND12-May-17ACTIVE5/12/17OTATOCTGTNot EnteredSL-701,
Multivalent Glioma-Associated Antigen Vaccine, Via Subcutaneous
InjectionNo product listed for IND 17492Emergency - Treatment of
glioblastoma in 4 patientsEMUFPCELL AND GENE THERAPY
17577IND28-Jun-17ACTIVE6/28/17OTATOCTGTNot EnteredAllogeneic
bone marrow-derived mesenchymal stem cellsNo product listed for IND
17577Emergency - primary progressive multiple sclerosisEM
17641IND1-Aug-17EM DENIED8/1/17OTATOCTGTNot EnteredAutologous
Adipose Derived Stromal Vascular Fraction and ACAM200 Vaccinia
VirusNo product listed for IND 17641Emergency - Treatment of
papillary thyroid carcinomaEMG1
17711IND7-Sep-17ACTIVE10/6/17OTATOCTGTCowan MD, Morton
JAutologous, bone marrow derived CD34+ cells transduced with
lentiviral vector, expressing the human cDNA for DCLRE1C (Artemis)
with the human Artemis promoterMultiple products listed for IND
17711Artemis-deficient severe combined immunodeficiency disease
(ARTSCID)RHG11NUFPCELL AND GENE THERAPY
17313IND6-Jan-17ACTIVE2/3/17OTATOCTGTKurtzberg MD,
JoanneAllogeneic human cord tissue-derived mesenchymal stromal
cells (hCT-MSC)Allogeneic human cord-tissue mesenchymal stromal
cells (hCT-MSC)Treatment of children with autism spectrum disorder
(ASD)RH1S3NUFPCELL AND GENE THERAPY
17512IND23-May-17EXEMPT6/22/17OTATOCTGTRizzieri MD, David
AG-CSF-mobilized peripheral blood stem cells (G-PBSCs)No product
listed for IND 17512Acute Myeloid LeukemiaRH3S5NUFPCELL AND GENE
THERAPY
17763IND5-Oct-17ACTIVE11/3/17OTATOCTGTMendell MD, Jerry
RrAAVrh74.MHCK7.Micro-Dystrophin, Non-Replicating, Recombinant
Adeno-Associated Virus, Seroype rh74 Containing a Human
Micro-Dystrophin Gene Controled by the Muscle-Specific MHCK7
PromoterrAAVs, seroype rh74 expressing human micro-dystrophin with
a muscle-specific [MHCK7] promoter
(rAAVrh74.MHCK7.Micro-dystrophin)Duchenne muscular
dystrophyRHG51NUFPCELL AND GENE THERAPY
17650IND1-Sep-17ACTIVE9/29/17OTATOCTGTBavarian Nordic
A/SVaccinia Virus Vector (BN-Brachyury) and Fowlpox Virus Vector
(FPV-Brachyury), Both Expressing Human Brachyury TAA, LFA-3, B7.1,
and ICAM-1 GenesMultiple products listed for IND 17650Treatment of
cancers that highly express Brachyury Tumor Associated Antigen
(lung, ovarian, breast, prostate, colorectal, pancreatic or
chordoma)CMG52NUFPCELL AND GENE THERAPY
17487IND10-May-17ACTIVE6/9/17OTATOCTGTCox Jr MD, Charles
SAutologous Umbilical Cord Blood Stem Cells for Individual Patient
Expanded AccessAutologous Cord Blood Stem CellsCerebral
palsyRHSPS3NUFPCELL AND GENE THERAPY
17396IND8-Mar-17ACTIVE4/6/17OTATOCTGTKrance MD, Robert
AAllogeneic Mesenchymal Stem CellsAllogeneic Bone Marrow Derived
Mesenchymal Stem CellsAplastic anemiaRHEMS2NUFPCELL AND GENE
THERAPY
17726IND4-Oct-17WITHDRAWN11/1/17OTATOBRRCSL BehringImmune
Globulin Intravenous (Human), Privigen (IgPro10)Immune Globulin
(Human)Chronic Immune Thrombocytopenic Purpura
(ITP)CM4NUFPHEMATOLOGICS
17350IND7-Feb-17ACTIVE2/28/17OTATOCTGTParney MD PhD, Ian
FAutologous Dendritic Cells Derived from (PBMC) CD14 Selection and
Pulsed with Allogeneic Tumor Lysate (ALTL-DCV)Multiple products
listed for IND 17350Single patient use for recurrent
glioblastomaRHSPS4NUFPCELL AND GENE THERAPY
17833IND15-Nov-17ACTIVE11/15/17OTATOCTGTTalano MD,
Julie-AnBPX-701No product listed for IND 17833Emergency - Treatment
of refractory AMLRHEMG4NUFPCELL AND GENE THERAPY
17625IND24-Jul-17ACTIVE8/23/17OTATOCTGTGillanders MD, William
ENeoantigen DNA Vaccine +/- DurvalumabMultiple products listed for
IND 17625Triple-Nagative Breast Cancer Disease following Standard
ChemotherapyRHG515UFPCELL AND GENE THERAPY
17596IND13-Jul-17ACTIVE8/10/17OTATOCTGTGillanders MD, William
ENeoantigen DNA VaccineMultiple products listed for IND
17596Pancreatic CancerRHG61NUFPCELL AND GENE THERAPY
17322IND3-Feb-17ACTIVE3/3/17OTATOCTGTAdvaxis IncLive Attenuated
Listeria monocytogenes Expressing Personalized Tumor AntigensLive
Attenuated Listeria monocytogenes Expressing Personalized Tumor
Antigenstreatment of metastatic cancerCMG81NUFPCELL AND GENE
THERAPY
17603IND17-Jul-17ACTIVE8/16/17OTATOCTGTZarour MD, Hassane MPD-1
responder (PD-1 R) derived Fecal Microbiota Transplantation (FMT) +
PembrolizumabMultiple products listed for IND 17603PD-1 resistant/
refractory advanced melanomaRH28UFPCELL AND GENE THERAPY
17796IND25-Oct-17HOLD11/22/17HOLD11/22/17OTATOCTGTShapiro MD,
Shane AAutologous Adipose-derived Stromal Vascular Fraction
(SVF)Autologous, uncultured adipose derived mononuclear
cellsAerodigestive FistulaeRH1NUFPCELL AND GENE THERAPY
17660IND11-Aug-17ACTIVE9/7/17OTATOCTGTWilliams MD, David
AAutologous CD34+ cells transduced ex vivo with Lentivirus
expressing BB694 LCD(-HS4) bGp D12G5 shmiRNo product listed for IND
17660Treatment of autologous CD34+ cells ex vivo for the treatment
of Sickle Cell DiseaseRHG11NUFPCELL AND GENE THERAPY
17679IND23-Aug-17ACTIVE9/22/17OTATOCTGTWilliams MD, David
AAutologous CD34+ cells transduced ex vivo with the lentiviral
vector rHIV-IL2RcoG2SCID (G2SCID)Lentiviral vector clinical
batchtreatment of x-linked severe combined immunodeficiency
syndromeRHG11NUFPCELL AND GENE THERAPY
17800IND26-Oct-17ACTIVE11/24/17OTATOCTGTCapricor IncAllogeneic
Unrelated, Cardiosphere-Derived CellsAllogeneic Cardiopshre-Derived
Cells (CDCs)Duchenne Muscular DystrophyCM2S3NORPHAN
DESIGNATIONUFPCELL AND GENE THERAPY
17439IND12-Apr-17ACTIVE5/12/17OTATOCTGTTransgene SAPexa-Vec
(pexastimogene devacirepvec, JX-594) vaccinia virus engineered to
express GM-CSF, and nivolumab, PD-1 receptor blocking
antibody.Vaccina/GM-CSF Virus Expressing GM-CSF GeneTreatment of
hepatocellular carcinomaCMG51NUFPCELL AND GENE THERAPY
17633IND28-Jul-17ACTIVE7/28/17OTATOCTGTRudnick MD,
JeremyAutologous Dendritic Cells Derived from Peripheral Blood
Mononuclear Cells, Expanded with Interleukin-4,
Granulocyte-Macrophage Colony-Stimulating Factor; Pulsed with
Synthetic Tumor-Stem Cell Associated Antigens [AIM-2, MAGE-1,
gp100, HER-2, IL-13Ra2] (ICT-107)utologous Dendritic Cells Derived
from Peripheral Blood Mononuclear CellsSingle Patient Use -
Treatment of glioblastoma in 3 patients.RHEMS4NUFPCELL AND GENE
THERAPY
17851IND21-Dec-17PENDING12/21/17OTATOCTGTNational Cancer
InstituteMVA-BN-Brachyury, M7824 (MSB001359C), ALT-803, and
Epacadostat (INCB024360)Multiple products listed for IND
17851Metastatic Castration Resistant Prostate CancerCMG518UFPCELL
AND GENE THERAPY
17764IND6-Nov-17ACTIVE12/6/17OTATOCTGTNational Cancer
InstitutePhase 1 Dose Escalation Study of CD19/CD22 Chimeric
Antigen Receptor (CAR) T Cells in Children and Young Adults with
Recurrent and Refractory CD19/CD22-expressing B Cell
Malignancies.No product listed for IND 17764Recurrent or
refractory, CD19/CD22-expressing, B cell acute lymphoblastic
leukemia or lymphomaCMG11NUFPCELL AND GENE THERAPY
17811IND20-Nov-17ACTIVE12/20/17OTATOCTGTNational Cancer
InstituteETBX-071 (adenoviral PSA vaccine); ETBX-061 (adenoviral
MUC1 vaccine); ETBX-051 (adenoviral brachyury vaccine)No product
listed for IND 17811Metastatic Castration Resistant Prostate
CancerCMG51NUFPCELL AND GENE THERAPY
17676IND2-Oct-17ACTIVE11/1/17OTATOCTGTNational Cancer
InstituteAdenovirus Vector Type 5 Expressing CEA Gene; Adenovirus
Vector Type 5 Expressing Muc1 Gene; and Adenovirus Vector Type 5
Expressing Brachyury GeneNo product listed for IND 17676Advanced
CancerCMG51NUFPCELL AND GENE THERAPY
17628IND23-Oct-17ACTIVE11/21/17OTATOCTGTNational Cancer
InstituteVaccinia Virus Vector (PROSTVAC-V), CV301 (MVA-BN-CV301)
and MSB0011359C) Expressing PSA, CEA, MUC-1, LFA-3, B7.1, and
ICAM-1Multiple products listed for IND 17628Biochemically Recurrent
Prostate CancerCMG526UFPCELL AND GENE THERAPY
17712IND11-Sep-17ACTIVE10/11/17OTATOCTGTInovio Pharmaceuticals
IncDNA Plasmid Vectors pGXI 108 (encodes Prostate-specific Membrane
Antigen (PSMA)), pGXl404 (encodes Wilms' tumor gene 1 (WT1)
antigen)), pGXl434 (encodes Human Telomerase Reverse Transcriptase
(hTERT)) and DNA Plasmid Vectors pGX6001 ((IL-12) p35 and p40
subunit proteins))Multiple products listed for IND 17712Treatment
of Bladder Cancer (Urothelial Cancer, UCa)CMG618UFPCELL AND GENE
THERAPY
17671IND8-Sep-17ACTIVE10/6/17OTATOCTGTInovio Pharmaceuticals
IncINO-5401 (DNA plasmids pGX1108, pGX1404, and pGX1434) and
INO-9012 (DNA plasmid pGX6001) delivered by electroporation using
CELLECTRA 2000 in combination with REGN2810 [Fully Human Monoclonal
Antibody to Programmed Death-1 (PD-1)]No product listed for IND
17671Treatment of Glioblastoma (GBM)CMG618UFPCELL AND GENE
THERAPY
17666IND31-Aug-17ACTIVE9/29/17OTATOBRRGrifols Therapeutics
IncImmune Globulin (Human)Immune Globulin (Human) Serum,
IntravenousPassive pre-exposure prophylaxis of
HAVCM4NUFPHEMATOLOGICS
17637IND13-Nov-17ACTIVE12/13/17OTATOCTGTReNeuron Group
PLCAllogeneic Human Neural Stem Cells (CTX0E03 DP) transduced with
a retroviral vector expressing myc with ReNeuron-specific delivery
device.Multiple products listed for IND 17637Treatment of patients
with stable, moderate or moderately severe residual disability
following an ischemic strokeCMG125UFPCELL AND GENE THERAPY
17324IND17-Jan-17ACTIVE2/16/17OTATOCTGTHare MD, Joshua
MAllogeneic Umbilical Cord Tissue derived Mesenchymal Stem Cells
(UCMSCs) and Bone Marrow derived Human Mesenchymal Stem Cells
(BMMSCs)Multiple products listed for IND 17324Cytokine suppression
in patients with chronic inflammation due to Metabolic
syndrome.RH1S3NUFPCELL AND GENE THERAPY
16897IND31-Jan-17ACTIVE4/11/17HOLD3/2/17OTATOBRRGreen Cross
CorpImmune Globulin Intravenous (Human), 10% (GC 5107)No product
listed for IND 16897For the treatment of primary humoral deficiency
(PHID)CM3NUFPHEMATOLOGICS
17449IND14-Apr-17ACTIVE5/12/17OTATOCTGTCairo MD, Mitchell SViral
Specific Cytotoxic T-CellsT-Cells, Allogeneic, Related Donor,
EBV-SpecificAdenovrius, Epstein-Barr Virus,
CytomegalovirusRH2S3NUFPCELL AND GENE THERAPY
17884IND14-Dec-17ACTIVE12/21/17OTATOCTGTCairo MD, Mitchell
SRemestemcel-L, allogeneic Mesenchymal Stromal CellsNo product
listed for IND 17884Steroid Refractory Acute GVHD in a single
patientRHSPS3NUFPCELL AND GENE THERAPY
17787IND20-Oct-17ACTIVE11/1/17OTATOCTGTCairo MD, Mitchell
SAllogeneic, Bone Marrow-Derived, Immuno-Selected, and Ex Vivo
Expanded Mesenchymal Precursor Cells, Intravenous Infusion
[Rexlemestrocel-L]Allogeneic, Bone Marrow-Derived, Immuno-Selected,
and Ex Vivo Expanded Mesenchymal Precursor Cells, Intravenous
Infusiontreatment of refractory junctional Epidermis Bullosa in a
single patientRHSP1S3NUFPCELL AND GENE THERAPY
17400IND23-Mar-17ACTIVE4/22/17OTATOCTGTBluebird Bio
IncAutologous T Lymphocyte-enriched Cells Tranduced with Lentiviral
Vector Encoding a Chimeric Antigen Receptor Targeting B Cell
Maturation Antigen (anti-BCMA02 CAR); Cultured with a
Phosphatidylinositol 3-kinase InhibitorNo product listed for IND
17400Treatment of multiple myeloma and lymphomaCMG11NRARE
DISEASEUFPCELL AND GENE THERAPY
17522IND1-Jun-17ACTIVE7/1/17OTATOCTGTBlock MD PhD, Matthew
SAutologous Dendritic Cells derived from PBMC pulsed with Prevnar
(Pneumococcal 13-valent Conjugate Vaccine [(Diphtheria CRM197
Protein),Pembrolizumab [MK-3475 (anti-PD-1 antibody), [Keytruda],
following Cryoablation TherapyAutologous Dendritic Cells derived
from PBMCImmunotherapy for metastatic unresectable
melanomaRH2S4NUFPCELL AND GENE THERAPY
17815IND7-Nov-17ACTIVE12/7/17OTATOCTGTCenter for Cell and Gene
TherapyAutologous Natural Killer T cells transduced with a
retroviral vector expressing GD2-specific chimeric antigen receptor
and interleukin-15 (GD2.CAR NKT cells)No product listed for IND
17815Relapsed and Refractory NeuroblastomaRHG11NUFPCELL AND GENE
THERAPY
17588IND5-Jul-17ACTIVE8/4/17OTATOCTGTCenter for Cell and Gene
TherapyTumor-Associated Antigen (TAA)-Specific Cytotoxic T
Lymphocytes (CTLs) (TACTOPS)No product listed for IND
17588Treatment of pancreatic cancer patients with metastatic or
locally advanced unresectable or resectable diseaseRH1S4NUFPCELL
AND GENE THERAPY
17586IND5-Jul-17ACTIVE8/4/17OTATOCTGTCenter for Cell and Gene
TherapyTumor-Associated Antigen (TAA)-Specific Cytotoxic T
Lymphocytes (CTLs)No product listed for IND 17586Treatment of
breast cancer patients with metastatic or locally recurrent
unresectable disease as measured by clinical benefit rate according
to RECIST criteriaRH1S4NUFPCELL AND GENE THERAPY
17461IND25-Apr-17ACTIVE5/25/17OTATOCTGTCenter for Cell and Gene
TherapyAutologous T-Cells Transduced with Retroviral Vectors
Expressing a second Generation Chimeric Antigen Receptor CD5.CARNo
product listed for IND 17461Relapsed/refractory CD5+ T cell
malignanciesRH1S3NUFPCELL AND GENE THERAPY
17391IND20-Mar-17ACTIVE4/19/17OTATOBRRCSL Behring LLCC1 Esterase
Inhibitor (Human)C1 Esterase Inhibitor, HumanChronic treatment of
refractory antibody mediated rejection (AMR) in adult kidney
transplant recipients in combination with i.v. immunoglobulin
containing regimensCM3NRARE DISEASEUFPCELL AND GENE THERAPY
17335IND1-Feb-17ACTIVE3/3/17OTATOCTGTBorrello MD,
IvanLenalidomide with or without Allogeneic Myeloma GM-CSF Vaccine
(Irradiated H929, U266 and K562/GM-CSF cells)Multiple products
listed for IND 17335Treatment of Multiple MyelomaRH2S4NUFPCELL AND
GENE THERAPY
17414IDE3-Mar-17REFUSE TO ACCEPT3/28/17OTATOCTGTTissue Genesis
LLCCell Isolation SystemFat (Adipose) TissuesTreatment of the
Symptoms of Erectile DysfunctionCM1NUFMDEVICES
17589IND6-Jul-17WITHDRAWN10/4/17OTATOCTGTThe Methodist Hospital
Research InstituteAutologous adipose-derived mesenchymal stem
cellsAutologous adipose-derived mesenchymal stem cellsTreatment of
Autoimmune Thromocytopenia Purpura and Hemolytic Uremic Syndrome in
a Single PatientRHSPS3NUFPCELL AND GENE THERAPY
17690IND28-Aug-17ACTIVE9/27/17OTATOCTGTChilds MD, Richard
WAutologous enriched T cells (CD8+/CD34+) Transduced with
Retroviral Vector (HERV-E TCR CD34t 7G1) Expressing HERV-E T cell
Receptor GeneNo product listed for IND 17690Clear cell Renal Cell
CarcinomaRHG4NUFPCELL AND GENE THERAPY
17348IND7-Feb-17ACTIVE3/9/17OTATOCTGTChilds MD, Richard
WAllogeneic Cord Blood-derived Ex Vivo Expanded with or without
Allogeneic Haploidentical Donor Co-transplantationMultiple products
listed for IND 17348Severe Aplastic Anemia (SAA) and
Myelodysplastic Syndrome (MDS)RH2S3NUFPCELL AND GENE THERAPY
17685IND25-Aug-17ACTIVE9/14/17OTATOCTGTBota MD PhD, Daniela
AAutologous Dendritic Cells Derived from Peripheral Blood
Mononuclear Cells, Expanded with Interleukin-4,
Granulocyte-Macrophage Colony-Stimulating Factor; Pulsed with
Synthetic Tumor-Stem Cell Associated Antigens [AIM-2, MAGE-1,
gp100, HER-2, IL-13Ra2] (ICT-107)Autologous Dendritic Cells Derived
from Peripheral Blood Mononuclear CellsSINGLE PATIENT USE:
Treatment for GlioblastomaRHSPS4NUFPCELL AND GENE THERAPY
17504IND1-Jun-17ACTIVE6/30/17OTATOCTGTGovindan MD,
RamaswamyPilot Feasibility Study of the Combination of a
Personalized Therapeutic Anti-tumor Vaccine with
PembrolizumabMultiple products listed for IND 17504Non-Small Cell
Lung CancerRH1NUFPCELL AND GENE THERAPY
17748IND1-Dec-17ACTIVE12/29/17OTATOCTGTJuno Therapeutics
IncAutologous CD4+ and CD8+ T Cells (JCARH125) Transduced with
Replication-Incompetent, Self-Inactivating (SIN) Lentiviral
Vector(v20008) encoding Chimeric Antigen Receptor (CAR-T) and
B-Cell Maturation Antigen (BCMA)Autologous CD4+ and CD8+ T cells
Transduced with Self-Inactivating (SIN) Lentiviral Vector (v20008)
Expressing CD19-specific Chimeric Antigen Receptor (CAR)
(JCAR0125)Immunotherapy for relapsed or refractory multiple
myelomaCMG11NORPHAN DESIGNATIONUFPCELL AND GENE THERAPY
17444IND12-Apr-17ACTIVE5/12/17OTATOCTGTMueller MD PhD,
SabineModified Measles virus MV-NISHuman Thyroidal Sodium-Iodide
Symporter Gene (Measles Virus Vector) MV-NISMultiply recurrent
medulloblastoma single patient expanded accessRHSPG5NUFPCELL AND
GENE THERAPY
17678IND23-Aug-17ACTIVE12/13/17HOLD9/22/17OTATOCTGTBleakley MD
PhD, MarieAllogenic CD8+ and CD4+ Memory T Cells Transduced with a
Lentiviral Vector (LV; pRRLSIN) Containing iC9-HA1 TCR2-RQR-CD8
transgene to Express an HA-1-Specific T cell ReceptorMultiple
products listed for IND 17678Patients with persistent or recurrent
acute leukemia undergoing or following HCTRHG41NUFPCELL AND GENE
THERAPY
17576IND28-Jun-17ACTIVE6/28/17OTATOCTGTLevy MD PhD, Michael
LAllogeneic-Unrelated Bone Marrow Derived Mesenchymal Stem CellsNo
product listed for IND 17576Emergency - Treatment of COPDEM
17584IND3-Jul-17ACTIVE12/15/17HOLD8/2/17OTATOCTGTWake Forest
Institute for Regenerative MedicineAutologous corporal smooth
muscle cells (SMC) and endothelia cells(EC) seeded on
decellularized corpora cavernose.Multiple products listed for IND
17584Repair or replace irreversibly damaged penile
corporaRH1S5NUFPCELL AND GENE THERAPY
17583IND3-Jul-17HOLD8/2/17HOLD8/2/17OTATOCTGTWake Forest
Institute for Regenerative MedicineAutologous Urothelial Cells (UC)
and Smooth Muscle Cells (SMC) Seeded on a Polyglycolic Acid (PGA)
Bladder Shaped ScaffoldMultiple products listed for IND
17583Subjects with Fibrotic Contracted BladderRH2S55UFPCELL AND
GENE THERAPY
17562IND22-Jun-17ACTIVE9/20/17HOLD7/21/17OTATOCTGTBollard MD,
CatherineAutologous ex vivo expanded HIV-1 specific T-cells
(HST-NEETs)No product listed for IND 17562Therapy in HIV-infected
individuals suppressed on antiretroviral therapy (ART)RH1S3NUFPCELL
AND GENE THERAPY
17762IND5-Oct-17ACTIVE10/11/17OTATOCTGTKiromic IncAutologous
Dendritic Cells Derived from Peripheral Blood Mononuclear Cells,
Cultured with Interleukin-4, Granulocyte-Macrophage
Colony-Stimulating Factor, Tumor Necrosis Factor-alpha,
Interleukin-1beta, Pulsed with Tumor Antigens and Followed by Low
Dose Interleukin-2Multiple products listed for IND
17762Hematological malignanciesCM1NUFPCELL AND GENE THERAPY
17669IND25-Aug-17ACTIVE9/24/17OTATOBRRCambium Medical
Technologies LLCFibrinogen-Depleted Human Platelet LysateMultiple
products listed for IND 17669Dry Eye Secondary to Graft vs. Host
DiseaseCM1NUFPHEMATOLOGICS
17482IND5-May-17EXEMPT5/16/17OTATOCTGTRamsingh MD,
GiridharanHLA-mismatched allogeneic cellular therapy
(Microtransplantation)No product listed for IND
17482Intermediate-Risk Acute Myeloid Leukemia