FT-001 / Frontera - LARVOL DELTA

Safety and Efficacy Results of an Open-Label, Dose-Escalation and Dose-Expansion Phase 1/2 Study of FT-001 in Subjects With RPE65-IRD (AAO 2024) - P1/2 | "In Phase 2, a significant improvement of MT score was observed among all five patients until Day 84, accompanied by improvement of FST result too. Conclusion FT-001 holds great promise as an effective treatment for patients with RPE65-IRD."

Clinical • P1/2 data • Ophthalmology • Retinal Disorders

Phase I study evaluating the safety, tolerability and preliminary efficacy of FT-001 gene therapy for RPE65 associated retinal dystrophy (ARVO 2024) - P1/2 | "FT-001 is generally tolerated with good safety profile, After one time's subretinal administration of FT-001, subjects' functional vison and light sensitivity were greatly improved since Week 4 and remained effective. FT-001 holds great promise to be an effective treatment for patients with RPE65 associated retinal dystrophy."

Clinical • Gene therapy • P1 data • Inherited Retinal Dystrophy • Ocular Inflammation • Ophthalmology • Retinal Disorders

Assessing the Potential of Small Peptides for Altering Expression Levels of the Iron-Regulatory Genes FTH1 and TFRC and Enhancing Androgen Receptor Inhibitor Activity in In Vitro Prostate Cancer Models. (PubMed, Int J Mol Sci) - "We examined the ability of LNCaP, PC3, VCaP, and VCaP-EnzR cells to form colonies in the presence of androgen receptor inhibitors (ARI) and a series of iron-gene modulating oligopeptides (FT-001-FT-008)...Colony growth suppression generally correlated with the magnitude of concurrent increases in FTH1 and decreases in TFRC expression for all cells. The results of this study provide preliminary insight into a novel approach at targeting iron dysmetabolism and sensitizing PCa cells to established cancer treatments."

Journal • Preclinical • Genito-urinary Cancer • Metabolic Disorders • Oncology • Prostate Cancer • Solid Tumor • FTH1

Antiproliferative activity of marine-derived oligopeptides combined with androgen receptor inhibition in preclinical prostate cancer models. (ASCO 2023) - " Eight candidate FTH1-modulatory peptides (FT-001 to -008, all 7- or 8-mer peptides) were identified in the SPH peptide mix and subsequently sequenced and synthesized. Antiproliferative effects of individual FT peptides were assessed in combination with ARis (bicalutamide/BIC or enzalutamide/ENZ) in phenotypically distinct PC cell lines (LNCaP, PC3, VCaP, ENZR-VCaP) and compared to the inhibitory effect of SPH-ARi combination or ARi monotherapy... FT-002 and -005 synergistically combine with ARis to significantly attenuate PC tumor growth in vitro. Gene expression profiling implicates peptide-mediated FTH1 and TFRC modulation in the mode of action to interfere with tumor cell iron metabolism. Novel research perspectives implicate ferritin and FTH1 expression levels in prostate carcinogenesis and tumor progression."

Preclinical • Genito-urinary Cancer • Metabolic Disorders • Oncology • Prostate Cancer • Solid Tumor • FTH1

Gene Therapy in Subjects With Biallelic RPE65 Mutation-associated Retinal Dystrophy (clinicaltrials.gov) - P1/2 | N=9 | Recruiting | Sponsor: Frontera Therapeutics

Gene therapy • New P1/2 trial • Gene Therapies • Inherited Retinal Dystrophy