gene therapy for arthritis / Precigen, Paragon Biosciences - LARVOL DELTA

AdenoVerse GT: A single therapeutic engineered for immune-evasion and sustained treatment of alpha-1 antitrypsin liver and lung disease (ASGCT 2026) - "Introduction Although seemingly straightforward, curative gene therapy for most monogenetic diseases remains elusive...To further improve potency and mitigate intracellular anti-vector responses, a CpG-reduced transgene cassette was evaluated and found to further increase AAT PiM expression from human hepatocytes as well as reduce liver enzyme activation and anti- capsid neutralizing antibody production in immune-competent mice. Conclusion Taken together, these results support advancement of the AdenoVerse GT vector for further development and application for other diseases in which liver-restricted expression is of interest."

Alpha-1 Antitrypsin Deficiency • Gene Therapies • Genetic Disorders • Hepatology • Pulmonary Disease • Respiratory Diseases • ELANE

PRGN-2009 and bintrafusp alfa for patients with advanced or metastatic human papillomavirus-associated cancer. (PubMed, Cancer Immunol Immunother) - P1/2 | "PRGN-2009 was well tolerated, and immune responses were observed to PRGN-2009. Encouraging anti-tumor activity and OS were noted in the combination with BA arm, consisting mainly of checkpoint-resistant patients. Trial Registration ClinicalTrials.gov Identifier: NCT04432597."

Journal • Cervical Cancer • Gene Therapies • Head and Neck Cancer • Oncology • Oropharyngeal Cancer • Solid Tumor • CXCL8 • TGFB1

PRGN-2012 gene therapy in adults with recurrent respiratory papillomatosis: a pivotal phase 1/2 clinical trial. (PubMed, Lancet Respir Med) - P1/2 | "PRGN-2012 treatment resulted in complete response in 51% of the patients treated and was safe. Based on these positive pivotal study results, a biologics license application to the US Food and Drug Administration (FDA) is planned, positioning PRGN-2012 to be an FDA-approved medical treatment for adult patients with RRP."

Journal • P1/2 data • Fatigue • Gene Therapies • Infectious Disease • Oncology

PRGN-2012, a novel gorilla adenovirus-based immunotherapy, provides the first treatment that leads to complete and durable responses in recurrent respiratory papillomatosis patients. (ASCO 2024) - P1/2 | "PRGN-2012 is a gorilla adenovirus-based gene therapy/immunotherapy designed to generate HPV6/11-specific T cell immunity. These data demonstrate the overall favorable safety profile and significant clinical benefit of PRGN-2012 in adult RRP patients. These findings support PRGN-2012 as a potential therapeutic option for this patient population where no FDA-approved therapeutics exist."

Clinical • Late-breaking abstract • Fatigue • Gene Therapies • Infectious Disease • Musculoskeletal Pain • Oncology • Pain

Improving Control of Gene Therapy-Based Neurotrophin Delivery for Inner Ear Applications. (PubMed, Front Bioeng Biotechnol) - "Hearing loss due to overexpression of neurotrophins in the normal hearing ear was avoided when using this novel vector-promoter combination. Combining supporting cell-specific gene delivery via the adenovirus serotype 28 vector with a low-strength long expressing promoter potentially can provide long-term neurotrophin delivery to the damaged inner ear."

Journal • Gene Therapies • Otorhinolaryngology • BDNF • NTF3

"$SGMO @SangamoTx @pfizer @AlbertBourla @biogen @sanofi #CNS #AAV #GENETHERAPY @ProfMakris @Precigen $PGEN" (,@Biotech2050)

Gene Therapies