Genespire Presents Positive Preclinical Proof of Concept Data at the ASGCT Annual Meeting
2024年5月10日 - 1:00AM
PRESS RELEASE
Genespire Presents Positive Preclinical
Proof of Concept Data at the ASGCT Annual Meeting
- Data provides preclinical validation for Genespire’s
liver-directed lentiviral gene therapy in a mouse model of
methylmalonic acidemia
- Abstract received the ASGCT Excellence In Research Award
Milan, Italy, 9 May 2024 –
Genespire, a biotechnology company developing off-the-shelf gene
therapies for pediatric patients affected by genetic diseases,
today presented positive preclinical data at the 27th Annual
Meeting of the American Society of Gene and Cell Therapy (ASGCT)
currently taking place in Baltimore, MD, USA. These data
demonstrated long-term preclinical efficacy of the Company’s
liver-directed integrative immune shielded lentiviral vector (ISLV)
based gene therapy in a mouse model of methylmalonic acidemia
(MMA).
MMA is a rare, genetic metabolic disorder most
frequently caused by a faulty gene coding for the mitochondrial
enzyme methylmalonyl-coA mutase (MUT). People with this condition
are unable to break down and use certain proteins and fats found in
food and, as a result, circulating methylmalonic acid accumulates
in the body, causing damage to the brain, liver, kidneys, and other
organs. At present there are no disease-targeted drugs approved for
MMA, and affected patients suffer high levels of morbidity and have
a heavily reduced life expectancy.
In the study presented at ASGCT, intravenous
administration of an ISLV encoding a human MUT transgene was
evaluated in a mouse model of MMA. Treated mice showed a rapid,
substantial, and long-lasting (>1 year) reduction in circulating
methylmalonic acid, normalization of weight and complete survival.
Additionally, ISLV administration led to a systemic effect,
resulting in a significant reduction of methylmalonic acid levels
not only in the liver but also in the brain and kidney, suggesting
an extrahepatic benefit achieved through liver-mediated
detoxification.
This work was performed by Dr. Elena Barbon,
post-doctoral fellow, and Dr. Alessio Cantore, Group Leader, at the
San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), in
collaboration with Genespire. Dr. Elena Barbon received the ASGCT
Excellence In Research Award for this abstract. This award is
granted by the ASGCT to the top 15 abstracts received from ASGCT
members or associate members.
Alessio Cantore, Group Leader at
SR-TIGET and co-founder of Genespire added: “These
compelling data provide evidence for the efficacy, safety, and
extrahepatic benefit of our integrative immune shielded lentiviral
vector (LV)-based gene therapy and important preclinical
validation.”
Julia Berretta, CEO at Genespire,
commented: “Receiving the ASGCT Excellence In Research
Award provides further external validation on the quality of our
research and promise of our technology”. She added, “We believe
that our unique ISLV gene therapy approach, allowing for stable
gene transfer and long-lasting expression of the therapeutic gene,
may be optimally positioned to treat MMA patients.”
For further information, please reach out to the Company.
-ENDS-
Enquiries:
Genespire |
Tel: +39 02
80896651
info@genespire.com |
|
|
ICR
Consilium |
Tel: +44 (0) 20 3709 5700 |
Amber Fennell/Ashley
Tapp/Jonathan Edwards |
Genespire@consilium-comms.com |
|
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About
GenespireGenespire is a biotechnology company, developing
off-the-shelf gene therapies based on immune shielded lentiviral
vectors (ISLVs) for pediatric patients affected by genetic
diseases. ISLVs are designed to be used intravenously and allow the
life-long production of the therapy directly from the patient’s
liver. Genespire is initially advancing therapeutic programs in
inherited metabolic diseases with high unmet medical need. Based in
Milan, Italy, Genespire was founded in March 2020 by the gene
therapy pioneer Prof. Luigi Naldini and Dr. Alessio Cantore,
Fondazione Telethon and Ospedale San Raffaele. Genespire is a
spin-out of SR-Tiget, a world leading cell and gene therapy
research institute. Find out more about us at
www.genespire.com.