Associated tags: Mortality, Patient, Muscle weakness, Neuromuscular disease, Respiratory failure, Disease, Life, MTM1, Astellas Pharma, Kate, DSM-IV codes, AT132, SAN, X-linked myotubular myopathy, Safety, TSE, Pharmaceutical industry
Astellas Pharma,
MTM1,
Kate,
DSM-IV codes,
AT132,
Muscle weakness,
Neuromuscular disease,
Patient,
SAN,
X-linked myotubular myopathy,
Safety,
Respiratory failure,
TSE,
Pharmaceutical industry TOKYO and SAN DIEGO, June 8, 2023 /PRNewswire/ -- Astellas Pharma Inc. (TSE: 4503, President and CEO: Naoki Okamura, "Astellas"), and Kate Therapeutics ("KateTx") today announced an exclusive license agreement to develop and commercialize KT430. KT430 is a preclinical next-generation investigational gene therapy that delivers a functional copy of the MTM1 gene via a novel MyoAAVÂ capsid to treat X-linked myotubular myopathy (XLMTM), a serious, life-threatening, rare neuromuscular disease characterized by extreme muscle weakness, respiratory failure and early death.
Key Points:
- TOKYO and SAN DIEGO, June 8, 2023 /PRNewswire/ -- Astellas Pharma Inc. (TSE: 4503, President and CEO: Naoki Okamura, "Astellas"), and Kate Therapeutics ("KateTx") today announced an exclusive license agreement to develop and commercialize KT430.
- Under the terms of the agreement, Astellas will make an undisclosed upfront payment to KateTx, which is also eligible to receive development, regulatory and commercial milestone payments, plus royalties on worldwide sales.
- Astellas will receive an exclusive worldwide license to develop, manufacture and commercialize KT430.
- "This agreement brings together Astellas and KateTx's collective patient-focused missions, allowing us to evaluate how to advance this new potential therapy for people diagnosed with XLMTM," stated Adam Pearson, Chief Strategy Officer at Astellas.
Retrieved on:
Saturday, October 5, 2019
Biotechnology,
Pharmaceutical,
Health,
FDA,
Genetics,
Clinical trials,
Aspiro,
Jay-Z,
X-linked Myotubular Myopathy,
AT132 for the treatment of X-linked Myotubular Myopathy,
Audentes Therapeutics, Inc. "The new ASPIRO data shared today builds upon the encouraging efficacy and safety profile seen to date with AT132, stated Dr. Dowling.
Key Points:
- "The new ASPIRO data shared today builds upon the encouraging efficacy and safety profile seen to date with AT132, stated Dr. Dowling.
- We remain focused on our goal of rapidly progressing AT132 toward global regulatory approvals, stated Natalie Holles, President and Chief Operating Officer of Audentes.
- The newly reported data include safety and efficacy assessments as of the August 7, 2019 data cut-off date for 12 patients enrolled in the ASPIRO dose escalation cohorts.
- Audentes is developing AT132, an AAV8 vector containing a functional copy of the MTM1 gene, for the treatment of XLMTM.
Retrieved on:
Monday, September 30, 2019
Health,
Genetics,
Clinical trials,
Pharmaceutical,
Biotechnology,
Rare diseases,
Centronuclear myopathy,
Myotubularin 1,
Jay-Z,
X-linked myotubular myopathy,
Myopathy,
Aspiro,
X-linked Myotubular Myopathy,
AT132 for the treatment of XLMTM,
Audentes Therapeutics, Inc. Dr. Dowling will present new efficacy and safety data from the ASPIRO Phase 1/2 dose escalation cohorts (10 treated patients and 2 controls).
Key Points:
- Dr. Dowling will present new efficacy and safety data from the ASPIRO Phase 1/2 dose escalation cohorts (10 treated patients and 2 controls).
- We are excited to share new efficacy and safety data from our ASPIRO study, stated Natalie Holles, President and Chief Operating Officer.
- Audentes is planning several additional presentations during the conference, including a company-sponsored symposium that will provide an in-depth review of XLMTM and the ASPIRO study results.
- Audentes is developing AT132, an AAV8 vector containing a functional copy of the MTM1 gene, for the treatment of X-linked Myotubular Myopathy (XLMTM).
