Cholestasis, Progressive Familial Intrahepatic 3

Vivet Therapeutics AnnouncesMultiple Projects Update, IncludingNew VTX-803 Preclinical data To Be PresentedAt 2021 American Society Of Gene And Cell Therapy AnnualMeeting Compelling additional preclinical data further support therapeutic potential of gene therapy product to treat Progressive Familial IntrahepaticCholestasis Type 3 (PFIC3). Paris, France, May 10, 2021 – Vivet Therapeutics (“Vivet”), a clinical stage biotech company dedicated to developing gene therapy treatments for inherited liver disorders with high unmet medical need, today announced that new data regarding VTX-803, Vivet’s AAV-based gene therapy in development for the treatment of PFIC3, will be presented during the ASGCT 24th Annual Meeting, May 11 - 14, 2021. New findings from the recently completed preclinical studies of VTX-803 will be presented by Dr. Nicholas D. Weber, Research Scientist at Vivet. VTX-803 was already shown to normalize key serum biomarkers, hepatosplenomegaly and liver fibrosis in two-week-old (pre-fibrotic liver) or five-week-old (fibrotic liver) PFIC3 mice. Dr Weber will present new animal data expanding those results: “AAV-Based Gene Therapy for PFIC3 (VTX-803) Shows Further Promise with Durable Efficacy in PFIC3 Mice at Different Disease Stages” (Tuesday May 11, 2021; 8:00-10:00 AM EST – Abstract 503). These data show VTX-803 therapeutic effect in older animals (16 weeks) presenting severely fibrotic/cirrhotic livers, without an increase in dose. Dr. Weber will also present VTX-803 long-term studies showing the vector to be safe and the effect to be durable at least up to 6 months and comparable between 5-week-old and 16-week-old mice. Dr. Gloria González-Aseguinolaza, Vivet Therapeutics CSO and Deputy Director of Cima center of the Fundación para la Investigación Médica Aplicada (FIMA) will co-chair a session: “Gene Therapy for Inborn Errors of Metabolism” (Tuesday May 11, 2021; 5:30-7:30 PM EST), where Dr. Nick Weber will also be presenting additional data on VTX-803. Vivet is also developing an AAV-based gene therapy product, VTX-802 for Progressive Familial Intrahepatic Cholestasis Type 2 (PFIC2), currently at preclinical stage. Vivet and Mirum Pharmaceuticals recently announced an exclusive worldwide option, collaboration and license agreement for Vivet’s Gene Therapy programs targeting Progressive Familial Intrahepatic Cholestasis, VTX-802 and VTX-803. In addition, compelling data from a new animal proof of concept study for VTX-804, one of Vivet’s portfolio indications, an AAV-based gene therapy for Citrullinemia Type 1 (CTLN-1) will be presented by Andrea Bazo, PhD Student at Cima center-FIMA: “rAAV-Mediated Gene Therapy in Combination with Short-Term Nitrogen-Scavenger Treatment Corrects Biochemical and Behavioral Abnormalities and Increases Lifespan in Infant Citrullinemia Type 1 (CTLN-1) Mice” (Tuesday May 11, 2021; 8:00-10:00 AM EST – Abstract 520). CTLN-1 is a rare autosomal recessive urea cycle defect characterized biologically by hyperammonemia and clinically by progressive lethargy, poor feeding and vomiting in the neonatal form and by variable hyperammonemia in the later-onset form patients. These preclinical data show that a single dose of VTX-804 administered intravenously to infantile CTLN-1 mice in association with Standard of Care (SOC) normalized lifespan and corrected biochemical and behavioral abnormalities up to 6 months post administration, making VTX-804 a potentially very promising therapeutic strategy for young CTLN-1 patients. The abstracts listed above can be found online at: About Vivet Therapeutics Vivet Therapeutics is a clinical stage emerging biotechnology company developing novel gene therapy treatments for rare, inherited metabolic diseases. Vivet is building a diversified gene therapy pipeline based on novel recombinant adeno-associated virus (rAAV) technologies developed through its partnerships with, and exclusive licenses from, the Fundación para la Investigación Médica Aplicada (FIMA), a not-for-profit foundation at the Centro de Investigación Medica Aplicada (CIMA), University of Navarra based in Pamplona, Spain. Vivet’s lead program, VTX-801, currently under IND clinical development with the GATEWAY clinical trial, is a novel investigational gene therapy for Wilson disease which has been granted Orphan Drug Designation (ODD) by the Food and Drug Administration (FDA) and the European Commission (EC). This rare genetic disorder is caused by mutations in the gene encoding the ATP7B protein, which reduces the ability of the liver and other tissues to regulate copper levels causing severe hepatic damage, neurologic symptoms and potentially death. Vivet’s second gene therapy product, VTX-803 for PFIC3, received US and European Orphan Drug Designation in May 2020. Vivet is supported by international life science investors including Novartis Venture Fund, Roche Venture Fund, HealthCap, Pfizer Inc., Columbus Venture Partners, Ysios Capital, Kurma Partners and Idinvest Partners. Please visit us at and follow us on Twitter at @Vivet_tx and LinkedIn. About PFIC Progressive Familial Intrahepatic Cholestasis (PFIC) is a rare genetic disorder that causes progressive liver disease typically leading to liver failure. In people with PFIC, liver cells are less able to secrete bile or essential components into the bile. The resulting dysregulation in the bile processing causes liver disease in affected individuals. Signs and symptoms of PFIC typically begin in infancy. Patients experience severe itching, jaundice, failure to grow at the expected rate (failure to thrive), and an increasing inability of the liver to function (liver failure). The disease is estimated to affect one in every 50,000 to 100,000 births in the United States and Europe. Six types of PFIC have been genetically identified, all of which are similarly characterized by impaired bile flow and progressive liver disease. The PFIC2 patient population accounts for approximately 60% of the PFIC patient population. PFIC2 is caused by a mutation in the ABCB11 gene, which normally encodes a bile salt export pump protein that moves bile acids out of the liver. PFIC3, is caused by a genetic defect in the ABCB4 gene (also designated MDR3) which encodes a phospholipid translocator involved in biliary phospholipid (phosphatidylcholine) excretion. In PFIC3, cholestasis results from the toxicity of bile in which detergent bile salts are not inactivated by phospholipids, leading to bile canaliculi and biliary epithelium injuries.

WATERTOWN, Mass., April 29, 2021 (GLOBE NEWSWIRE) -- Selecta Biosciences, Inc. (NASDAQ: SELB), a biotechnology company leveraging its clinically validated ImmTOR™ platform to develop tolerogenic therapies that selectively mitigate unwanted immune responses, today announced three upcoming presentations at the 24th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT), to be held virtually from May 11-14, 2021. These presentations further demonstrate the potential of Selecta’s ImmTOR platform to mitigate AAV immunogenicity and enable redosing of gene therapy treatments for various genetic disorders. “We are excited to present data on ImmTOR’s potential to address current limitations in the gene therapy field including efficacy, safety and durability at ASGCT,” said Dr. Takashi Kei Kishimoto, Ph.D., chief scientific officer of Selecta. “The results to be presented continue to highlight ImmTOR’s ability to enable redosing of AAV-based gene therapies and prevent the production of AAV-specific neutralizing antibodies (NAbs) in vivo. As many gene therapies will be targeted to pediatric patients, maintaining therapeutic activity is of the utmost importance to families of patients with genetic disorders, a concern that we believe ImmTOR has the potential to solve moving forward.” Oral Presentation:Presentation Title: Coadministration of AAV Expressing MDR3 (VTX-803) and ImmTOR Allows for Vector Re-Administration to Treat Progressive Familial Intrahepatic Cholestasis Type 3 (PFIC3) in Juvenile Abcb4-/- MiceSession Title: Gene Therapy for Inborn Errors of MetabolismAbstract Number: 29Presenter: Nicholas D. Weber, Ph.D. Presentation Date and Time: Tuesday, May 11, 2021 5:30 p.m. ET An oral presentation from scientists at Vivet Therapeutics will highlight the potential of ImmTOR to enable repeated intravenous administration of liver-directed AAV vector carrying human ABCB4 cDNA (VTX-803) in a juvenile mouse model of progressive familial intrahepatic cholestasis type 3 (PFIC3). These data highlight the power of redosing AAV gene therapy with ImmTOR to sustain therapeutic efficacy in juvenile animals. Digital Poster Presentations:Presentation Title: ImmTOR Nanoparticles Promote Survival and Enable Repeat Gene Therapy of MMUT-Deficient Mice with Maternally-Transferred Anti-AAV AntibodiesSession Title: Metabolic, Storage, Endocrine, Liver and Gastrointestinal DiseasesAbstract Number: 483Presenter: Petr Ilyinskii, Ph.D.Presentation Date and Time: Tuesday, May 11, 2021 8 a.m. ET Results presented in this poster show the potential of a combination of ImmTOR and AAV-based gene therapy vector to mitigate the detrimental impact of maternally-transferred anti-AAV antibodies in a mouse model of methylmalonic acidemia (MMA). Presentation Title: ImmTOR Nanoparticles Enhance the Level and Durability of AAV Transgene Expression after Initial Dosing and Mitigate the Formation of Neutralizing Antibodies in Nonhuman PrimatesSession Title: Immunological Aspects of Gene Therapy and VaccinesAbstract Number: 761Presenter: Takashi K. Kishimoto, Ph.D.Presentation Date and Time: Tuesday, May 11, 2021 8 a.m. ET This poster presents data from a large NHP study that builds upon previous data showing that tolerogenic ImmTOR nanoparticles can selectively mitigate anti-AAV T and B cell responses to enable vector redosing in mice and small nonhuman primates (NHP). These results indicate that ImmTOR may enhance the level and durability of transgene expression after initial treatment of AAV vector while inhibiting the formation of neutralizing antibodies. Following the conference, the three presentations will be available in the Resources section of Selecta’s website at . About Selecta Biosciences, Inc.Selecta Biosciences Inc. (NASDAQ: SELB) is leveraging its clinically validated ImmTOR™ platform to develop tolerogenic therapies that selectively mitigate unwanted immune responses. With a proven ability to induce tolerance to highly immunogenic proteins, ImmTOR has the potential to amplify the efficacy of biologic therapies, including redosing of life-saving gene therapies, as well as restore the body’s natural self-tolerance in autoimmune diseases. The company’s first program aimed at addressing immunogenicity to AAV gene therapies is expected to enter clinical trials in early 2021 in partnership with AskBio for the treatment of methylmalonic acidemia (MMA), a rare metabolic disorder. A wholly-owned program focused on addressing IgA nephropathy driven by ImmTOR and a therapeutic enzyme is also in development among additional product candidates. Selecta recently licensed its Phase 3 clinical product candidate, SEL-212, in chronic refractory gout to Sobi. For more information, please visit . Selecta Forward-Looking StatementsAny statements in this press release about the future expectations, plans and prospects of Selecta Biosciences, Inc. (“the company”), including without limitation, statements regarding the unique proprietary technology platform of the company, and the unique proprietary platform of its partners, the potential of ImmTOR to enable re-dosing of AAV gene therapy, the potential treatment applications of product candidates utilizing the ImmTOR platform in areas such as gene therapy, the ability of the Company and AskBio to develop gene therapy products using ImmTOR and AskBio’s technology, the novelty of treatment paradigms that the Company is able to develop, whether the observations made in non-human primate study subjects will translate to studies performed with human beings, the potential of any therapies developed by the company and AskBio to fulfill unmet medical needs, the company’s plan to apply its ImmTOR technology platform to a range of biologics for rare and orphan genetic diseases, the potential of the company’s intellectual property to enable repeat administration in gene therapy product candidates and products, the ability to re-dose patients and the potential of ImmTOR to allow for re-dosing, the potential to safely re-dose AAV, the ability to restore transgene expression, the potential of the ImmTOR technology platform generally and the company’s ability to grow its strategic partnerships,  and other statements containing the words “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “hypothesize,” “intend,” “may,” “plan,” “potential,” “predict,” “project,” “should,” “target,” “would,” and similar expressions, constitute forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including, but not limited to, the following: the uncertainties inherent in the initiation, completion and cost of clinical trials including proof of concept trials, including the uncertain outcomes, the availability and timing of data from ongoing and future clinical trials and the results of such trials, whether preliminary results from a particular clinical trial will be predictive of the final results of that trial or whether results of early clinical trials will be indicative of the results of later clinical trials, the ability to predict results of studies performed on human beings based on results of studies performed on non-human primates, the unproven approach of the company’s ImmTOR technology, potential delays in enrollment of patients, undesirable side effects of the company’s product candidates, its reliance on third parties to manufacture its product candidates and to conduct its clinical trials, the company’s inability to maintain its existing or future collaborations, licenses or contractual relationships, its inability to protect its proprietary technology and intellectual property, potential delays in regulatory approvals, the availability of funding sufficient for its foreseeable and unforeseeable operating expenses and capital expenditure requirements, the company’s recurring losses from operations and negative cash flows from operations raise substantial doubt regarding its ability to continue as a going concern, substantial fluctuation in the price of its common stock, and other important factors discussed in the “Risk Factors” section of the company’s most recent Quarterly Report on Form 10-Q, and in other filings that the company makes with the Securities and Exchange Commission. In addition, any forward-looking statements included in this press release represent the company’s views only as of the date of its publication and should not be relied upon as representing its views as of any subsequent date. The company specifically disclaims any intention to update any forward-looking statements included in this press release. For Investors:Bruce MackleLifeSci Advisors, LLC+1-929-469-3859bmackle@lifesciadvisors.com For Media: Meredith Sosulski, Ph.D.LifeSci Communications, LLC+1-929-469-3851msosulski@lifescicomms.com