{"id":22892,"date":"2023-05-24T09:24:57","date_gmt":"2023-05-24T07:24:57","guid":{"rendered":"https:\/\/www.izb-online.de\/praeklinische-wirksamkeits-und-sicherheitsdaten-fuer-vigenerons-innovativen-ansatz-zur-behandlung-von-retinitis-pigmentosa-innovations-und-gruenderzentrum-biotechnologie-izb\/"},"modified":"2023-05-24T14:29:55","modified_gmt":"2023-05-24T12:29:55","slug":"preclinical-efficacy-and-safety-data-for-vigenerons-innovative-approach-to-the-treatment-of-retinitis-pigmentosa-innovations-und-gruenderzentrum-biotechnologie-izb","status":"publish","type":"post","link":"https:\/\/www.izb-online.de\/en\/izb-biotech-news\/preclinical-efficacy-and-safety-data-for-vigenerons-innovative-approach-to-the-treatment-of-retinitis-pigmentosa-innovations-und-gruenderzentrum-biotechnologie-izb\/","title":{"rendered":"Preclinical efficacy and safety data for ViGeneron\u2019s innovative approach to the treatment of Retinitis Pigmentosa"},"content":{"rendered":"

[vc_row el_class=”gco-magazine-container”][vc_column el_class=”bildspalte”][vc_row_inner][vc_column_inner offset=”vc_col-lg-12 vc_col-md-12″][vc_column_text el_class=”gco-magazine–title”]<\/p>\n

Preclinical efficacy and safety data for ViGeneron\u2019s innovative approach to the treatment of Retinitis Pigmentosa<\/h1>\n

[\/vc_column_text][vc_column_text el_class=”gco-magazine-lead”]The company\u2019s lead program VG901 is the first gene therapy targeting CNGA1 mutation of this hereditary eye disease.[\/vc_column_text][vc_column_text el_class=”gco-magazine–post-date”]May, 2023[\/vc_column_text][\/vc_column_inner][\/vc_row_inner][vc_row_inner el_class=”two-columns” el_id=”corona-ausblenden”][vc_column_inner el_class=”bild-zwei-spalten-links”][vc_single_image image=”6283″ img_size=”full”][vc_column_text el_class=”gco-magazine-copyright”]\u00a9 Pieris[\/vc_column_text][vc_column_text el_class=”gco-magazine-picture-discription”]The Lungs[\/vc_column_text][vc_single_image image=”6285″ img_size=”full”][vc_column_text el_class=”gco-magazine-copyright”]\u00a9 Pieris[\/vc_column_text][vc_column_text el_class=”gco-magazine-picture-discription”]Stephen S. Yoder<\/strong>, CEO, Pieris Pharmaceuticals Inc.[\/vc_column_text][\/vc_column_inner][\/vc_row_inner][vc_row_inner][vc_column_inner][vc_single_image image=”22888″ img_size=”full”][vc_column_text el_class=”gco-magazine-copyright”]\u00a9 Shutterstock \/ Kateryna Kon[\/vc_column_text][vc_column_text el_class=”gco-magazine-picture-discription”]Retinitis pigmentosa, a genetic eye disease. 3D image shows the pigment deposits in the retina, the attenuated blood vessels, the pigmentary bone marrow spicules and the waxy appearance of the optic disc.[\/vc_column_text][\/vc_column_inner][\/vc_row_inner][vc_row_inner el_class=”gco-magazine–quote” css=”.vc_custom_1611310713611{margin-top: 33px !important;}”][vc_column_inner][vc_column_text]<\/p>\n

\u201cOur VG901 program, which uses vgAAV vector to deliver the CNGA1 gene via intravitreal injection, has demonstrated promising preclinical data. This represents a significant step towards providing a novel, first-in-class therapy for patients born with CNGA1 mutations and potentially saving their eyesight as they age.\u201d<\/h6>\n

Dr. Caroline Man Xu, Co-Founder and CEO of ViGeneron<\/p>\n

[\/vc_column_text][\/vc_column_inner][\/vc_row_inner][vc_row_inner el_class=”gco-magazine-container–copy” css=”.vc_custom_1591795890737{margin-top: 21px !important;}”][vc_column_inner][vc_column_text]IZB-alumnus ViGeneron GmbH, a next-generation gene therapy company, presented its VG901 program for CNGA1-associated retinitis pigmentosa (RP) in an oral presentation at the American Society of Gene & Cell Therapy (ASGCT) Annual Meeting in Los Angeles from May 16-23, 2023. VG901 is a gene supplementation therapy that uses vgAAV, ViGeneron\u2019s proprietary adeno-associated virus (AAV) vector, to deliver the CNGA1 gene via intravitreal injection. The presentation featured in vitro and in vivo proof-of-concept efficacy data and details on the GLP (Good Laboratory Practice) safety study.<\/p>\n

Dr. Caroline Man Xu, Co-founder, and CEO of ViGeneron said: \u201cAs a next-generation gene therapy company, ViGeneron is committed to developing innovative treatments for patients in need. Our VG901 program, which uses vgAAV vector to deliver the CNGA1 gene via intravitreal injection, has demonstrated promising preclinical data. This represents a significant step towards providing a novel, first-in-class therapy for patients born with CNGA1 mutations and potentially saving their eyesight as they age. We look forward to advancing this program into clinical development and providing a potential cure for patients who currently have no treatment options.\u201d<\/p>\n

Autosomal recessive retinitis pigmentosa (arRP) is a hereditary disease, with approximately 2% to 8% of all arRP cases being attributed to genetic defects in the CNGA1 gene. Loss of CNG channels in rod photoreceptors due to CNGA1 mutations leads to dysfunction and a slow decrease in cone function. The preclinical data shows that VG901 has the potential to supplement the CNGA1 gene in a mouse model of RP. Furthermore, a GLP-safety study applying a single intravitreal injection followed by a 6-month post treatment observation period confirmed the safety of VG901.<\/p>\n

About Retinitis pigmentosa (RP)
\n<\/strong>Retinitis pigmentosa (RP) is a group of related eye disorders that cause progressive vision loss. RP initially presents as night-time blindness during childhood and early adulthood, progressing to peripheral visual field loss and \u201ctunnel vision\u201d, central visual impairment, reduced visual acuity, in most cases the disease progresses to a complete loss of vision. It is estimated that 50-60% of RP are inherited autosomal recessively. Mutations in the CNGA1 gene, encoding a subunit of CNG channels in rod photoreceptors, are reported to cause approximately 2% \u2013 8% of autosomal recessive retinitis pigmentosa (arRP).<\/p>\n

ViGeneron develops two novel next-generation gene therapy platforms
\n<\/strong>ViGeneron is dedicated to bringing gene therapy innovations to people in need. The company is advancing its proprietary gene therapy pipeline to treat ophthalmic diseases, while partnering with leading biopharmaceutical players in retinal diseases, CNS, and other disease areas. ViGeneron\u2019s three novel next-generation gene therapy platforms are geared towards addressing the limitations of existing adeno-associated virus (AAV)-based gene therapies. The first, vgAAV vector platform, enables a superior transduction efficiency of target cells and is designed to overcome biological barriers, thus enabling novel, less invasive routes of administration such as intravitreal injections. The second, REVeRT (REconstitution Via mRNA Trans-splicing) technology platform, allows efficient reconstitution of large genes (>5Kb) in various tissues such as retina, brain, heart, liver, and skeletal muscle. The third, AAV Transactivation is a CRISPR-Cas\u2013based AAV gene therapy platform that enables to regulate one or multiple genes in vivo. Privately-owned ViGeneron was founded in 2017 by a seasoned team with in-depth experience in AAV vector technology and clinical ophthalmic gene therapy programs and is located in Munich, Germany. For further information, please visit\u00a0www.vigeneron.com<\/a>.[\/vc_column_text][vc_column_text css=”.vc_custom_1583761043671{background-color: #1e73be !important;}” el_class=”redaktionell”][\/vc_column_text][\/vc_column_inner][\/vc_row_inner]\n\n

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