BASEL, Switzerland I December 12, 2023 I Versameb AG (“Versameb”), a pre-clinical stage company focused on transforming RNA therapeutics to treat unmet medical needs, today announced the publication of a scientific manuscript in the internationally recognized scientific journal, Molecular Therapy – Nucleic Acids.
The paper, entitled ‘Local application of engineered insulin-like growth factor I mRNA demonstrates regenerative therapeutic potential in vivo‘, highlights recent findings, which further demonstrate the therapeutic potential of engineered IGF-I mRNA as a regenerative medicine with a favorable safety profile.
The study demonstrated that the local administration of engineered IGF-I mRNA, which ensures desirable homeostatic kinetics and non-systemic, local dose-dependent expression of IGF-I protein, can overcome the unfavorable pharmacokinetic profile of IGF-I which has previously prevented it from being used therapeutically. IGF-I deficiency is associated with several neurological and musculoskeletal diseases due to impaired growth and regeneration. The novel and proprietary technology used to engineer the heterologous signal peptide improved in vitro protein secretion and accelerated in vivo functional regeneration (16-fold) over endogenous IGF-I mRNA, while pre-clinical models confirmed that locally administered IGF-I mRNA remained at the site of injection, contributing to a favorable safety profile. These findings combine to demonstrate the therapeutic potential of engineered IGF-I mRNA and its clinical translatability in different diseases.
Dr Justin Antony Selvaraj, Technology Director of Versameb and first author of the publication, said: “Our study established the therapeutic benefit of engineered IGF-I mRNA in both “acute” muscle injury model and “chronic” spinal disc herniation model. These studies further confirm that locally administered mRNA did not distribute systemically and no local toxicity was observed, rendering a favorable safety profile.”.
Dr Klaas Zuideveld, Chief Executive Officer of Versameb, said: “These positive results further validate the potential of mRNA as a safe, regenerative therapeutic for the treatment of Stress Urinary Incontinence (SUI), a high unmet medical need affecting more than 30% of adult women globally with no current drug treatment approved. Our lead candidate, VMB-100, has demonstrated promising pre-clinical data showing muscle tissue regeneration and function restoration of the urinary sphincter and is commencing clinical development in H1 2024, following IND clearance received by the FDA in November 2023. We look forward to providing further details of our first-in-class mRNA therapy in the future as we transform the treatment paradigm for SUI.”
Citation:
Justin S. Antony, Pascale Birrer, Claudia Bohnert, Sina Zimmerli, Petra Hillmann, Hervé Schaffhauser, Christine Hoeflich, Andreas Hoeflich, Ramzi Khairallah, Andreas T. Satoh, Isabelle Kappeler, Isabel Ferreira, Klaas P. Zuideveld, and Friedrich Metzger. Local application of engineered insulin-like growth factor I mRNA demonstrates regenerative therapeutic potential in vivo. Molecular Therapy – Nucleic Acids, Volume 34, 2023, 102055. DOI: https://doi.org/10.1016/j.omtn.2023.102055
About Versameb
Versameb AG is a privately held biotechnology company focusing on discovering and developing innovative RNA-based drugs for modulation of protein expression, including the ability to simultaneously influence several therapeutic targets, in a controlled manner, with one molecular construct, and cellular targeting. Based in Basel and fully operational since 2018, the company is led by an experienced scientific and leadership team with proven expertise in drug discovery and development from lab bench to patient. Versameb’s proprietary technology platform, VERSagile, optimizes the application of functional RNA in different disease contexts. The pipeline includes lead candidate programs in stress urinary incontinence (SUI), solid tumors and rare diseases. Versameb is working towards the completion of a first in-human proof-of-concept clinical study while advancing its platform. More information on Versameb can be found at www.versameb.com as well as on LinkedIn.
SOURCE: Versameb
Post Views: 526
BASEL, Switzerland I December 12, 2023 I Versameb AG (“Versameb”), a pre-clinical stage company focused on transforming RNA therapeutics to treat unmet medical needs, today announced the publication of a scientific manuscript in the internationally recognized scientific journal, Molecular Therapy – Nucleic Acids.
The paper, entitled ‘Local application of engineered insulin-like growth factor I mRNA demonstrates regenerative therapeutic potential in vivo‘, highlights recent findings, which further demonstrate the therapeutic potential of engineered IGF-I mRNA as a regenerative medicine with a favorable safety profile.
The study demonstrated that the local administration of engineered IGF-I mRNA, which ensures desirable homeostatic kinetics and non-systemic, local dose-dependent expression of IGF-I protein, can overcome the unfavorable pharmacokinetic profile of IGF-I which has previously prevented it from being used therapeutically. IGF-I deficiency is associated with several neurological and musculoskeletal diseases due to impaired growth and regeneration. The novel and proprietary technology used to engineer the heterologous signal peptide improved in vitro protein secretion and accelerated in vivo functional regeneration (16-fold) over endogenous IGF-I mRNA, while pre-clinical models confirmed that locally administered IGF-I mRNA remained at the site of injection, contributing to a favorable safety profile. These findings combine to demonstrate the therapeutic potential of engineered IGF-I mRNA and its clinical translatability in different diseases.
Dr Justin Antony Selvaraj, Technology Director of Versameb and first author of the publication, said: “Our study established the therapeutic benefit of engineered IGF-I mRNA in both “acute” muscle injury model and “chronic” spinal disc herniation model. These studies further confirm that locally administered mRNA did not distribute systemically and no local toxicity was observed, rendering a favorable safety profile.”.
Dr Klaas Zuideveld, Chief Executive Officer of Versameb, said: “These positive results further validate the potential of mRNA as a safe, regenerative therapeutic for the treatment of Stress Urinary Incontinence (SUI), a high unmet medical need affecting more than 30% of adult women globally with no current drug treatment approved. Our lead candidate, VMB-100, has demonstrated promising pre-clinical data showing muscle tissue regeneration and function restoration of the urinary sphincter and is commencing clinical development in H1 2024, following IND clearance received by the FDA in November 2023. We look forward to providing further details of our first-in-class mRNA therapy in the future as we transform the treatment paradigm for SUI.”
Citation:
Justin S. Antony, Pascale Birrer, Claudia Bohnert, Sina Zimmerli, Petra Hillmann, Hervé Schaffhauser, Christine Hoeflich, Andreas Hoeflich, Ramzi Khairallah, Andreas T. Satoh, Isabelle Kappeler, Isabel Ferreira, Klaas P. Zuideveld, and Friedrich Metzger. Local application of engineered insulin-like growth factor I mRNA demonstrates regenerative therapeutic potential in vivo. Molecular Therapy – Nucleic Acids, Volume 34, 2023, 102055. DOI: https://doi.org/10.1016/j.omtn.2023.102055
About Versameb
Versameb AG is a privately held biotechnology company focusing on discovering and developing innovative RNA-based drugs for modulation of protein expression, including the ability to simultaneously influence several therapeutic targets, in a controlled manner, with one molecular construct, and cellular targeting. Based in Basel and fully operational since 2018, the company is led by an experienced scientific and leadership team with proven expertise in drug discovery and development from lab bench to patient. Versameb’s proprietary technology platform, VERSagile, optimizes the application of functional RNA in different disease contexts. The pipeline includes lead candidate programs in stress urinary incontinence (SUI), solid tumors and rare diseases. Versameb is working towards the completion of a first in-human proof-of-concept clinical study while advancing its platform. More information on Versameb can be found at www.versameb.com as well as on LinkedIn.
SOURCE: Versameb
Post Views: 526
