(AGENPARL) – Fri 26 September 2025 Lund, Sweden, 26 September, 2025. Hansa Biopharma AB, “Hansa” (Nasdaq Stockholm:
HNSA), today announced that Genethon will present preliminary efficacy and
safety data from the first patient treated in the GNT-018-IDES Phase 2 trial of
imlifidase and GNT-0003 in severe Crigler-Najjar syndrome at the European
Society of Gene and Cell Therapy (ESGCT) 32[nd] annual congress. On Thursday 9
October, between 8:30 AM and 10:30 AM CEST, Giuseppe Ronzitti, PhD, from
Genethon will present pre-clinical data from the Genethon-Hansa collaboration.
 The clinical data will be presented by Jeremy Do Cao, MD, co-investigator of
the clinical trial and paediatrician at Antoine-Béclère Hospital, AP-HP, Paris,
France on Friday 10 October, between 9:00 AM and 10:30 AM CEST.
GNT-018-IDES is a single-arm Phase 2 trial sponsored by Genethon evaluating the
efficacy and safety of a single intravenous administration of Genethon’s gene
therapy GNT-0003 following pre-treatment with imlifidase. In this trial, Hansa’s
first-in-class immunoglobulin G (IgG) antibody cleaving enzyme therapy is being
evaluated in patients with severe Crigler-Najjar syndrome and pre-existing
antibodies to AAV serotype 8 (AAV8). The trial is set to enroll a total of three
patients with severe Crigler-Najjar syndrome, pre-existing anti-AVV8 antibodies
and a requirement for prolonged daily phototherapy.
Renée Aguiar-Lucander, CEO, Hansa Biopharma said, “We look forward to the data
from the first patient treated in the GNT-018-IDES trial that Genethon will
present at ESGCT. This is an important step as we continue to gather evidence on
the potential of imlifidase in enabling gene therapy, where presence of anti-AAV
antibodies continues to be a barrier that precludes up to 1 in 3 patients from
being eligible to receive these treatments.”
Speaker Abstract Title Presentations details
Giuseppe Ronzitti, Genethon INV37: Harnessing the Thursday 9 October,
potential of 8:30 AM – 10:30 AM
Immunoglobulin G CEST.
degrading enzymes SESSION 7a: Immune
(Ide) for the Responses to GT
treatment of AAV
-seropositive
patients (https://www.
esgctcongress.com/prog
ramme-thu)
Jeremy Do Cao, Antoine OR086: Overcoming Friday 10 October,
-Béclère Hospital, AP-HP, AAV8 Immunity: First 9:00 AM – 10:30 AM
France Seropositive Crigler CEST.
-Najjar Patient SESSION 11a: Metabolic
Treated with GNT0003 Diseases II
Following Imlifidase
Pretreatment (GNT-018
-IDES clinical
trial) (https://www.es
gctcongress.com/progra
mme-fri)
— ENDS —
Contacts for more information:
Evan Ballantyne, Chief Financial Officer
Kerstin Falck, VP Global Corporate Affairs
Notes to editors
About imlifidase
Imlifidase is a unique antibody-cleaving enzyme originating from Streptococcus
pyogenes that specifically targets IgG and inhibits IgG-mediated immune
response.[1] It has a rapid onset of action, cleaving IgG-antibodies and
inhibiting their activity within hours after administration. Imlifidase has
conditional marketing approval in Europe and is marketed under the trade name
IDEFIRIX[®] for the desensitization treatment of highly sensitized adult kidney
transplant patients with a positive crossmatch against an available deceased
donor.[1]
About Crigler-Najjar syndrome
Crigler-Najjar syndrome is a rare genetic liver disease characterized by
abnormally high levels of bilirubin in the blood (hyperbilirubinemia), which
leads to irreversible neurological damage manifested as muscle weakness,
lethargy, deafness, mental retardation, and eye movement paralysis. This
accumulation of bilirubin is caused by a deficiency of the UGT1A1 enzyme,
responsible for transforming bilirubin into a substance that can be eliminated
by the body. It can result in significant neurological damage and death if not
treated quickly. At present, patients must undergo prolonged daily phototherapy
(often more than 10 hours a day, sometimes up to 15 hours a day) to keep their
bilirubin levels below the toxicity threshold. Crigler-Najjar syndrome is an
ultra-rare disease affecting less than one case per one million people per year.
Liver transplantation remains the only definitive cure to date, but is
associated with significant morbidity and mortality, as well as graft
shortage.[2]
About imlifidase and gene therapy
Imlifidase is currently being evaluated as a pre-treatment to gene therapy in
areas of high unmet need. Many gene therapies are based on the use of Adeno
Associated Viruses (AAV) vectors.[3-5] In some patients the immune system
carries antibodies that counteract the gene therapy treatment preventing its
success.[ 4-10] Pre-treatment with imlifidase prior to AAV-based gene therapy
treatment has the potential to inactivate antibodies and thereby enable gene
therapy in patients with pre-existing antibodies to AAV-based gene therapies.[9]
Currently, it is estimated that anti-AVV antibodies on average prevent 1 in 3
people from benefiting from gene therapy treatments.[ 4-7]
About Hansa Biopharma
Hansa Biopharma AB is a pioneering commercial-stage biopharmaceutical company on
a mission to develop and commercialize innovative, lifesaving and life-altering
treatments for patients with rare immunological conditions. The company has a
rich and expanding research and development program based on its proprietary IgG
-cleaving enzyme technology platform, to address serious unmet medical needs in
autoimmune diseases, gene therapy and transplantation. The company’s portfolio
includes imlifidase, a first-in-class immunoglobulin G (IgG) antibody-cleaving
enzyme therapy, which has been shown to enable kidney transplantation in highly
sensitized patients and HNSA-5487, a next-generation IgG cleaving molecule with
redosing potential. Hansa Biopharma is based in Lund, Sweden, and has operations
in Europe and the U.S. The company is listed on Nasdaq Stockholm under the
ticker HNSA. Find out more at http://www.hansabiopharma.com and follow us on
LinkedIn (https://www.linkedin.com/company/hansa-medical-ab/).
©2025 Hansa Biopharma AB. Hansa Biopharma, the beacon logo, IDEFIRIX, and
IDEFIRIX flower logo are trademarks of Hansa Biopharma AB, Lund, Sweden. All
rights reserved.
References
1. European Medicines Agency. Idefirix[®] summary of product characteristics.
Available at: https://www.ema.europa.eu/en/documents/product
-information/idefirix-epar-product-information_en.pdf.
2. https://www.genethon.com/our-pipeline/crigler-najjar-syndrome/. Last
accessed: August 2025
3. Lundstrom K. Viral Vectors in Gene Therapy: Where Do We Stand in 2023?
4. Boutin S, et al. Prevalence of serum IgG and neutralizing factors against
adeno-associated virus (AAV) types 1, 2, 5, 6, 8, and 9 in the healthy
population: implications for gene therapy using AAV vectors. Hum Gene Ther. 2010
5. Calcedo R, Wilson JM. Humoral Immune Response to AAV. Front Immunol. 2013
6. Veron P, Leborgne C, Monteilhet V, Boutin S, Martin S, Moullier P, Masurier
C. Humoral and cellular capsid-specific immune responses to adeno-associated
virus type 1 in randomized healthy donors. J Immunol. 2012 Jun 15;188(12):6418
7. Kruzik A, et al. Prevalence of Anti-Adeno-Associated Virus Immune Responses
in International Cohorts of Healthy Donors. Mol Ther Methods Clin Dev. 2019 Jun
8. Falese L, et al. Strategy to detect pre-existing immunity to AAV gene
therapy. Gene Ther. 2017 Dec;24(12):768-778. doi: 10.1038/gt.2017.95. Epub 2017
9. Leborgne C, et al. IgG-cleaving endopeptidase enables in vivo gene therapy
in the presence of anti-AAV neutralizing antibodies. Nat Med. 2020
Jul;26(7):1096-1101. doi: 10.1038/s41591-020-0911-7. Epub 2020 Jun 1. PMID:
10. Au H.K, et al. (2022) Gene Therapy Advances: A Meta-Analysis of AAV Usage in
Clinical Settings. Front. Med. 8:809118. doi: 10.3389/fmed.2021.809118
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Hansa Biopharma AB, Scheelevägen 22, Lund, 22362 Sweden
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