AAV Gene Therapy for Treating Congenital Hereditary Endothelial Dystrophy associated with Biallelic SLC4A11 Mutations

  • Post author:
  • Post category:

Translational Candidate Therapeutic candidate rAAV8-EF1α-hSLC4A11 is a recombinant AAV vector with single-stranded cDNA encoding the wild-type human SLC4A11 protein. Area of Impact The candidate is for treatment of congenital hereditary…

Continue ReadingAAV Gene Therapy for Treating Congenital Hereditary Endothelial Dystrophy associated with Biallelic SLC4A11 Mutations

Escape-Resistant Oligonucleotide Therapy (ONT) for Cytomegalovirus (CMV) Disease in Hematopoietic Stem-Cell and Solid-Organ Transplant Patients

  • Post author:
  • Post category:

Translational Candidate Cytomegalovirus antiviral FD-86: DNA Oligonucleotide Therapy Area of Impact HSCT transplant rejection and childhood cognitive and hearing impairment caused by Cytomegalovirus Mechanism of Action The candidate oligonucleotide therapy,…

Continue ReadingEscape-Resistant Oligonucleotide Therapy (ONT) for Cytomegalovirus (CMV) Disease in Hematopoietic Stem-Cell and Solid-Organ Transplant Patients

Development of a Gene Therapy for the Treatment of Pitt Hopkins Syndrome (PHS) – Translating from Animal Proof of Concept to Support Pre-IND Meeting

  • Post author:
  • Post category:

Translational Candidate MZ-1866 is a recombinant AAV9 based gene therapy containing the transgene encoding Transcription Factor 4 (TCF4) Area of Impact Pitt Hopkins Syndrome is a rare genetic neurological disease…

Continue ReadingDevelopment of a Gene Therapy for the Treatment of Pitt Hopkins Syndrome (PHS) – Translating from Animal Proof of Concept to Support Pre-IND Meeting

A Phase I/IIa Study to Evaluate the Efficacy of Toca 511/Toca FC with Standard of Care Therapy in Newly Diagnosed High Grade Glioma

  • Post author:
  • Post category:

Therapeutic Candidate or Device DB107 (Toca 511), a retroviral replicating vector expressing yeast cytosine deaminase, which converts an antifungal prodrug to an anticancer drug Indication Newly diagnosed high-grade glioma Therapeutic…

Continue ReadingA Phase I/IIa Study to Evaluate the Efficacy of Toca 511/Toca FC with Standard of Care Therapy in Newly Diagnosed High Grade Glioma

A Phase 2 Study Evaluating the Efficacy and Safety of IV Administered rAAV9 Vector Containing RP-A501 in Male Patients with Danon Disease

  • Post author:
  • Post category:

Therapeutic Candidate or Device RP-A501, a recombinant Adeno-Associated Virus Serotype 9 containing the LAMP2B transgene Indication Danon Disease Therapeutic Mechanism The proposed product, RP-A501, is an in vivo gene therapy…

Continue ReadingA Phase 2 Study Evaluating the Efficacy and Safety of IV Administered rAAV9 Vector Containing RP-A501 in Male Patients with Danon Disease

Personalized antisense oligonucleotide therapy for rare pediatric genetic disease: SCN2A

  • Post author:
  • Post category:

Therapeutic Candidate or Device Investigational personalized antisense oligonucleotide drug (nL-SCN2A-002) Indication SCN2a-associated genetic disorder Therapeutic Mechanism The study participant has a pathogenic de novo p.R853Q gain-of-function amino acid substitution mutation…

Continue ReadingPersonalized antisense oligonucleotide therapy for rare pediatric genetic disease: SCN2A