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  • ||||||||||  RGFP966 / BioMarin
    Preclinical, Journal:  HDAC3 inhibition protects against peripheral and central alterations in an animal model of obesity. (Pubmed Central) -  Sep 12, 2023   
    As preliminary preclinical toxicology analysis also suggests CP-8 as safe, our data endorse CP-8 as a novel anticancer molecule for treating cancers sensitive to homologous recombination-mediated DNA repair inhibitors. Therefore, HDAC3 inhibition can represent a promising pharmacological target for the treatment of obesity.
  • ||||||||||  Talzenna (talazoparib) / Pfizer
    Journal, Epigenetic controller:  SETD2 Deficiency Confers Sensitivity to Dual Inhibition of DNA Methylation and PARP in Kidney Cancer. (Pubmed Central) -  Sep 11, 2023   
    We tested the effects of the DNA HMA 5-aza-2'-dexoxydytidine (DAC), the PARPi talazoparib (BMN-673), and both in combination in human ccRCC models with or without SETD2 deficiency...Finally, the combination effectively suppressed the growth of SETD2-deficient ccRCC in in vivo mouse models. Together, these findings indicate that combining HMA and PARPi is a promising potential therapeutic strategy for treating SETD2-compromised ccRCC.
  • ||||||||||  Talzenna (talazoparib) / Pfizer, navitoclax (ABT 263) / AbbVie
    Journal, PARP Biomarker, PD(L)-1 Biomarker, IO biomarker:  Construction and validation of a novel ferroptosis-related prognostic signature for lung adenocarcinoma. (Pubmed Central) -  Sep 11, 2023   
    Lipid peroxide assay showed that silencing PRDX6 or ACSL3 promoted lipid peroxidation and ferroptosis in lung cancer cells. Our novel ferroptosis-related signature shows potential clinical and functional importance in LUAD patients, and further research on ferroptosis as a therapeutic target in LUAD is warranted.
  • ||||||||||  RGFP966 / BioMarin, tasquinimod (ABR-215050) / Active Biotech, Ipsen, Cognex (tacrine) / Shionogi
    Review, Journal:  Enhancement of neural regeneration as a therapeutic strategy for Alzheimer's disease (Review). (Pubmed Central) -  Aug 24, 2023   
    However, further evaluations need to be performed to determine the optimal treatment. The present study reviews recent interventions for enhancing adult neurogenesis and attempts to elucidate their mechanisms of action, which may provide a theoretical basis for inducing nerve regeneration to fight against AD.
  • ||||||||||  Roctavian (valoctocogene roxaparvovec) / BioMarin, Hemgenix (etranacogene dezaparvovec) / CSL Behring
    Review, Journal, Gene therapy:  Hemophilia Gene Therapy: First, Do No Harm. (Pubmed Central) -  Aug 21, 2023   
    Bias on the part of healthcare professionals and people with hemophilia must be addressed and minimized. Here, we review the data leading to the regulatory authorization in Europe of valoctocogene roxaparvovec, an adeno-associated virus (AAV)-5 gene therapy to treat hemophilia A and etranacogene dezaparvovec-drlb in the United States and Europe to treat hemophilia B. We also provide an overview of the decision-making process and recommend steps that should be taken by the hemophilia community to ensure the safety and optimal outcomes for people with hemophilia who choose to receive a gene therapy product.
  • ||||||||||  Talzenna (talazoparib) / Pfizer
    Trial completion date, Trial primary completion date, Combination therapy:  Talazoparib and Radiation Therapy in Treating Patients With Locally Recurrent Gynecologic Cancers (clinicaltrials.gov) -  Aug 15, 2023   
    P1,  N=24, Recruiting, 
    Here, we review the data leading to the regulatory authorization in Europe of valoctocogene roxaparvovec, an adeno-associated virus (AAV)-5 gene therapy to treat hemophilia A and etranacogene dezaparvovec-drlb in the United States and Europe to treat hemophilia B. We also provide an overview of the decision-making process and recommend steps that should be taken by the hemophilia community to ensure the safety and optimal outcomes for people with hemophilia who choose to receive a gene therapy product. Trial completion date: Dec 2022 --> Oct 2024 | Trial primary completion date: Dec 2022 --> Oct 2024
  • ||||||||||  Palynziq (pegvaliase-pqpz) / BioMarin
    Trial completion date, Trial primary completion date:  Preliminary Study of Brain Effects of Palynziq-Related Changes in Phenylalanine in Individuals With PKU (clinicaltrials.gov) -  Aug 14, 2023   
    P=N/A,  N=13, Enrolling by invitation, 
    Trial completion date: Dec 2022 --> Oct 2024 | Trial primary completion date: Dec 2022 --> Oct 2024 Trial completion date: Apr 2023 --> Apr 2024 | Trial primary completion date: Apr 2023 --> Apr 2024
  • ||||||||||  Aldurazyme (laronidase) / BioMarin, Sanofi
    Trial completion:  China Post-marketing Surveillance (PMS) Study of Aldurazyme (clinicaltrials.gov) -  Aug 14, 2023   
    P4,  N=12, Completed, 
    Trial completion date: Apr 2023 --> Apr 2024 | Trial primary completion date: Apr 2023 --> Apr 2024 Active, not recruiting --> Completed
  • ||||||||||  Talzenna (talazoparib) / Pfizer
    Journal:  Talazoparib (Talzenna) for prostate cancer. (Pubmed Central) -  Aug 9, 2023   
    A single IV infusion of BMN 293 to MYBPC3-/- mice resulted in early and sustained reduction in left ventricular hypertrophy and durable improvements in diastolic function. No abstract available
  • ||||||||||  Talzenna (talazoparib) / Pfizer
    Trial termination, Combination therapy, Tumor mutational burden, IO biomarker:  Javelin BRCA/ATM: Avelumab Plus Talazoparib in Patients With BRCA or ATM Mutant Solid Tumors (clinicaltrials.gov) -  Aug 7, 2023   
    P2,  N=202, Terminated, 
    Active, not recruiting --> Terminated; The study was terminated since there was no need for further safety or efficacy data to be collected. The participants having benefit from the investigational treatments have been moved to a continuation study (NCT05059522)
  • ||||||||||  Palynziq (pegvaliase) / BioMarin
    Non-Alcoholic Steatohepatitis in a Young Non-Obese Patient With Phenylketonuria (Exhibit Hall) -  Jul 29, 2023 - Abstract #ACG2023ACG_4434;    
    Case Description/ A 21-year-old female (BMI 28) with a past medical history of PKU previously on pegvaliase, Hashimoto thyroiditis, and depression presented after one-week of non-bilious non-bloody emesis...Trichrome stain highlights prominent perivenous perisinusoidal, periportal and bridging fibrosis and probable cirrhosis. (Panel B)
  • ||||||||||  Preclinical, Journal:  Efficacy of combined targeted therapy with PI3K and CDK4/6 or PARP and WEE1 inhibitors in neuroblastoma cell lines. (Pubmed Central) -  Jul 25, 2023   
    Moreover, combining MK?1775 and BMN673 decreased the numbers of cells in S phase to a greater extent than either drug alone, while when combining PD?0332991 and BYL719 the observed effect was close to that of PD?0332991 alone. To summarize, PI3K and CDK4/6 or PARP and WEE1 exhibited synergistic anti?NB effects and lower doses of the inhibitors could be utilized, thereby potentially reducing adverse side effects.
  • ||||||||||  Roctavian (valoctocogene roxaparvovec) / BioMarin, Hemgenix (etranacogene dezaparvovec) / CSL Behring
    Review, Journal, Gene therapy:  Evaluating Gene Therapy as a Potential Paradigm Shift in Treating Severe Hemophilia. (Pubmed Central) -  Jul 25, 2023   
    Long-term monitoring of efficacy, durability of gene expression, and safety are ongoing. Gene therapy presents a promising new therapeutic option for patients with hemophilia and warrants continued innovation and investigation.
  • ||||||||||  ART6043 / Artios Pharma
    Enrollment open, Monotherapy, Metastases:  Study of ART6043 in Advanced/Metastatic Solid Tumors Patients (clinicaltrials.gov) -  Jul 13, 2023   
    P1/2,  N=250, Recruiting, 
    These findings suggest that combining EGFR and Notch signalling blockade can potentially increase the response to PARP blockade. Not yet recruiting --> Recruiting
  • ||||||||||  Talzenna (talazoparib) / Pfizer, Opdivo (nivolumab) / Ono Pharma, BMS
    Enrollment closed, Enrollment change, Trial completion date, Trial primary completion date, Combination therapy, Tumor mutational burden, Metastases:  Nivolumab in Combination With Talazoparib in Melanoma and Mutations in BRCA or BRCA-ness Genes (clinicaltrials.gov) -  Jul 12, 2023   
    P2,  N=8, Active, not recruiting, 
    Not yet recruiting --> Recruiting Recruiting --> Active, not recruiting | N=37 --> 8 | Trial completion date: Jan 2024 --> Oct 2024 | Trial primary completion date: Jan 2024 --> Oct 2023
  • ||||||||||  Palynziq (pegvaliase) / BioMarin
    OPAL: A multicenter, observational study to evaluate the real-world outcomes of pegvaliase in adults with Phenylketonuria (Exhibitions Hall (ICC)) -  Jul 11, 2023 - Abstract #SSIEM2023SSIEM_707;    
    Further research is required with an improved PheCheck system to enhance the control of blood Phe levels, ease the burden on PKU patients and/or caregivers, and reduce the complications associated with PKU. The results of this ongoing study could provide meaningful insight into the real- world use of pegvaliase and help characterize patient outcomes associated with Phe reduction.