Gene therapy to tackle organ fibrosis

OUR COMPANY

Telomere Therapeutics is a gene therapy company developing disease modifying treatments for unmet medical needs, pioneering the repair of telomeres to treat organ fibrosis and other telomere-dysfunction syndromes for which there are no curative treatments.

Our lead program is an AAV-based therapy primarily focused on idiopathic pulmonary fibrosis (iPF), a disease with very poor prognosis and a high mortality rate, estimated at <5 years after diagnosis.

Thus far our results demonstrate, for the first time ever, the capacity to revert disease in animal model*. Our goal is to make this drug a reality for iPF patients.

*Ref: Povedano JM, Martinez P, Serrano R, Tejera Á, Gómez-López G, Bobadilla M, Flores JM, Bosch F, Blasco MA. Therapeutic effects of telomerase in mice with pulmonary fibrosis induced by damage to the lungs and short telomeres. Elife. 2018 Jan 30;7:e31299. doi: 10.7554/eLife.31299.

MEET OUR FOUNDERS

María A. Blasco

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Scientific director of the Oncology National Research Center of Oncology (CNIO) and head of the Telomeres & Telomerase Research Group (CNIO). Maria A. Blasco is considered a world reference in the field of molecular biology, she is devoted to the study of telomeres, telomerase and their role in cancer and ageing. Her work has been published in high impact journals such as Cell, Nature and Science among others.

Blasco achievements have been recognised through international awards: The European Association of Cancer Research “Young Investigator Award”, the Joseph Steiner Award, the EMBO gold medal, the Rey Jaime I Prize for Basic Research, the Körber European Science Award, the Spanish National Research Award in Biology Santiago Ramón y Cajal as well as the Premio Jaume I.

Blasco has received four Doctorate Honoris Causa in Spain, has been editor of several scientific journals as well as member of different national and international Scientific Committees. In 2020, she became a member of the Board of Trustees of Museo del Prado (Madrid, Spain). In 2022, she became a member of the Board of Trustees and President of the Scientific Advisory Council of the ICAR Foundation (International Centre for Ageing Research), as well as a member of the Advisory Board of FECYT.

Fàtima Bosch I Tubert

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Professor of Biochemistry and Molecular Biology and Director of the Center of Animal Biotechnology and Gene Therapy (CBATEG) at the Universitat Autònoma de Barcelona. Bosch received her PhD in biochemistry and molecular biology and her undergraduate degree in pharmacy from the University of Barcelona. She conducted postdoctoral studies at Vanderbilt University, Case Western Reserve University, and the NCI-Frederick Cancer Research Center.

Bosch has received international recognition through several awards: Rey Juan Carlos I, Narcís Monturiol, Cruz de “Sant Jordi”, Alberto Sols, ICREA Academia, Jacob's Ladder Norman Saunders International Research Prize. She was a founding member of the European Society of Gene and Cell Therapy and President of the Spanish Society of Gene and Cell Therapy.

Bosch is also a member of the Gene Doping Expert Group of the World Anti-Doping Agency and scientific advisory board member in several biotech companies. Her research has focused for more than 30 years on developing AAV-mediated gene therapy approaches for both, rare and highly prevalent diseases, including inherited metabolic and neurodegenerative disorders among others. Bosch has authored >180 scientific publications and 28 patents, most already transferred to biotech and pharma industry.

PULMONARY FIBROSIS

Idiopathic Pulmonary Fibrosis - The facts

One of the most common and deadly interstitial lung disease.

Unfortunately, there is currently no cure for iPF, although certain medicines may slow the progression of disease.

Primarily occurs in adults.

Lung transplant is the standard of care, yet applicable to less than 5% of patients with severe iPF.

The molecular cause of disease is unknown (idiopathic).

The bottom line: median survival of <5 years after diagnosis.

PIPELINE

PRODUCT

INDICATION

DISCOVERY

PRECLINICAL PoC

IND - ENABLING

CLINICAL

AAV-TERT-01

IPF

DISCOVERY

PRECLINICAL PoC

IND - ENABLING

CLINICAL

AAV-TERT-02

Undisclosed

DISCOVERY

PRECLINICAL PoC

IND - ENABLING

CLINICAL

AAV-TERT-03

Undisclosed

DISCOVERY

PRECLINICAL PoC

IND - ENABLING

CLINICAL

AAV-TERT-04

Undisclosed

DISCOVERY

PRECLINICAL PoC

IND - ENABLING

CLINICAL

INVESTORS

Esta empresa está capitalizada por Innvierte, iniciativa de inversión de CDTI, E.P.E. This company is capitalized by Innvierte, an investment program of CDTI, E.P.E.

INSTITUTIONS

Telomere Therapeutics ha sido beneficiaria de un proyecto subvencionado por el CDTI y apoyado por el Ministerio de Ciencia e Innovación y cofinanciado por la Unión Europea – Next Generation EU. Este proyecto pretende impulsar el desarrollo de una terapia génica pionera utilizando un vector viral adenoasociado (AAV) proporcionando la expresión de telomerasa en tejidos adultos, denominada terapia génica TERT, para el tratamiento de la Fibrosis Pulmonar Idiopática (FPI). Concretamente, el tratamiento en desarrollo consiste en una terapia génica basada en un AAV con una secuencia optimizada del gen TERT para la expresión de la telomerasa transcriptasa y elongamiento telómerico específicamente en células alveolares del epitelio pulmonar.

Proyecto con expediente CPP2021-008483 financiado por MCIN/AEI/ 10.13039/501100011033 y por la Unión Europea NextGenerationEU/ PRTR