Selecta Biosciences and AskBio Initiate First-in-Human Dose-Escalation Study to Evaluate ImmTOR in Gene Therapy – PRNewswire
Posted: February 19, 2021 at 5:52 am
WATERTOWN, Mass., and Research Triangle Park, N.C., Feb. 17, 2021 /PRNewswire/ --Selecta Biosciences, Inc. (NASDAQ: SELB) and Asklepios BioPharmaceutical, Inc. (AskBio), a wholly owned and independently operated subsidiary of Bayer AG, today announced the initiation of a Phase 1 dose-escalation trial of SEL-399, an adeno-associated viral serotype 8 (AAV8) empty vector capsid (EMC-101) containing no DNA combined with ImmTOR. The trial aims to determine the optimal dose of ImmTOR to mitigate the formation of antibodies to AAV8 capsids used in gene therapies.
"We are pleased to further evaluate ImmTOR's ability to reduce the formation of antibodies to AAV capsids and potentially enable gene therapy redosing by having initiated this dose-escalation study of SEL-399," said Carsten Brunn, Ph.D., president and chief executive officer of Selecta. "This trial builds upon our strong preclinical data in non-human primates and marks the first time that ImmTOR in conjunction with an AAV capsid has been dosed in humans, which is a significant milestone. Data from this study will inform the design of future clinical trials in patients as we seek to unlock the full potential of gene therapy."
The dose-escalation trial of SEL-399 is designed to evaluate the safety and preliminary efficacyof ImmTOR in gene therapy. The study, being conducted in healthy volunteers at the SGS Life Sciences Clinical Pharmacology Unit in Antwerp, Belgium, plans to enroll up to 45 subjects to investigate increasing doses of ImmTOR and EMC-101. Subjects will be randomized in a 3:1 ratio of ImmTOR plus empty AAV8 capsid to empty capsid alone. Preliminary efficacy will be measured by assessing levels of AAV8-specific neutralizing antibodies.
Jude Samulski, Ph.D., chief scientific officer and co-founder of AskBio said, "By determining the dose at which ImmTOR is able to inhibit the formation of AAV-specific antibodies,this study could be a significant first step toward overcoming some of the unwanted immune responses associated with gene therapies. We look forward to using these findings to inform future studies as we work to develop strategies for repetitive dosing of AAV, thus extending durability of expression."
Selecta and AskBio expect to report initial results from this clinical trial in the fourth quarter of 2021.
AboutSelecta 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 visitwww.selectabio.com.
About AskBioAsklepios BioPharmaceutical, Inc. (AskBio), a wholly owned and independently operated subsidiary of Bayer AG acquired in 2020,is a fully integrated AAV gene therapy company dedicated to developing life-saving medicines that cure genetic diseases. The company maintains a portfolio of clinical programs across a range of neuromuscular, central nervous system, cardiovascular and metabolic disease indications with a clinical-stage pipeline that includes therapeutics for Pompe disease, Parkinson's disease and congestive heart failure, as well as out-licensed clinical indications for hemophilia and Duchenne muscular dystrophy. AskBio's gene therapy platform includes Pro10, an industry-leading proprietary cell line manufacturing process, and an extensive AAV capsid and promoter library. With global headquarters in Research Triangle Park, North Carolina, and European headquarters in Edinburgh, UK, the company has generated hundreds of proprietary third-generation AAV capsids and promoters, several of which have entered clinical testing. Founded in 2001 and an early innovator in the gene therapy field, the company holds more than 500 patents in areas such as AAV production and chimeric and self-complementary capsids. Learn more atwww.askbio.comor follow us onLinkedIn.
About BayerBayer is a global enterprise with core competencies in the life science fields of health care and nutrition. Its products and services are designed to benefit people by supporting efforts to overcome the major challenges presented by a growing and aging global population. At the same time, the Group aims to increase its earning power and create value through innovation and growth. Bayer is committed to the principles of sustainable development, and the Bayer brand stands for trust, reliability and quality throughout the world. In fiscal 2019, the Group employed around 104,000 people and had sales of43.5 billion euros. Capital expenditures amounted to2.9 billion euros, R&D expenses to5.3 billion euros. For more information, visit http://www.bayer.com.
AskBio Forward-Looking StatementsThis press release contains "forward-looking statements." Any statements contained in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Words such as "believes," "anticipates," "plans," "expects," "will," "intends," "potential," "possible" and similar expressions are intended to identify forward-looking statements. These forward-looking statements include without limitation statements regarding AskBio's pipeline of development candidates; AskBio's collaboration with Selecta; AskBio's clinical trials, including its ability to enroll subjects, the timing of any such trials and any potential side effects; whether ImmTOR will be able to reduce the formation of antibodies to AAV capsids and potentially enable gene therapy redosing; the timing of and results from the SEL-399/101 trial; whether the SEL-399/101 study could be a significant first step in overcoming the immunogenicity concerns associated with gene therapies; AskBio's strategies for repetitive dosing of AAV, extending durability of expression; AskBio's goal of developing life-saving medicines aimed at curing genetic diseases; and the potential benefits of AskBio's development candidates to patients. These forward-looking statements involve risks and uncertainties, many of which are beyond AskBio's control. Known risks include, among others: AskBio may not be able to execute on its business plans and goals, including meeting its expected or planned regulatory milestones and timelines, clinical development plans and bringing its product candidates to market, due to a variety of reasons, including the ongoing COVID-19 pandemic, possible limitations of company financial and other resources, manufacturing limitations that may not be anticipated or resolved in a timely manner, potential disagreements or other issues with our third-party collaborators and partners, and regulatory, court or agency feedback or decisions, such as feedback and decisions from the United States Food and Drug Administration or the United States Patent and Trademark Office. Any of the foregoing risks could materially and adversely affect AskBio's business and results of operations. You should not place undue reliance on the forward-looking statements contained in this press release. AskBio does not undertake any obligation to publicly update its forward-looking statements based on events or circumstances after the date hereof.
Selecta Forward-Looking StatementsAny statements in this press release about the future expectations, plans and prospects ofSelecta 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 theSecurities 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.
SOURCE Asklepios BioPharmaceutical, Inc.
Original post:
Selecta Biosciences and AskBio Initiate First-in-Human Dose-Escalation Study to Evaluate ImmTOR in Gene Therapy - PRNewswire
- This Pitt program is leveling up the gene therapy workforce in Pittsburgh - University of Pittsburgh - March 12th, 2024
- Opinion | Banning Gain-of-Function Research Would Do Far More Harm Than Good - Medpage Today - March 12th, 2024
- The Biggest Challenges Facing Broader Adoption of Novel Cell and Gene Therapies - Pharmaceutical Executive - March 12th, 2024
- Research and development areas of focus - Bristol Myers Squibb - March 4th, 2024
- Overcoming the challenges of gene therapy - Drug Discovery News - March 4th, 2024
- Breakthrough Gene Therapy for LHON: Vision Restoration in Focus at Medical Conference - BNN Breaking - March 4th, 2024
- Revolutionizing Healthcare: Global Gene Therapy Market Surges to US$ 3.44 Billion by 2022 As Revealed In Ne... - WhaTech - March 4th, 2024
- Looking to the future of clinical trials: Gene therapy, precision medicine, and the ongoing quest for rare disease solutions - pharmaphorum - March 4th, 2024
- Complement system response to AAV vector gene therapy - EurekAlert - February 23rd, 2024
- 10 gene therapy companies you should know about - Labiotech.eu - February 23rd, 2024
- Fetal surgery expert Tippi MacKenzie shifts to in-utero gene therapy - STAT - February 23rd, 2024
- Rejuvenate Bio Announces Gene Therapy-Mediated Partial Reprogramming Extends Lifespan and Reverses Age ... - Business Wire - February 23rd, 2024
- Combatting HIV using gene therapy - Drug Target Review - February 23rd, 2024
- Expanding the Horizons of Cell and Gene Therapy - RegMedNet - February 23rd, 2024
- Global Advanced Therapy Medicinal Products CDMO Market Analysis Report 2024-2030: : Increasing Investment and ... - Yahoo Finance UK - February 23rd, 2024
- 2023 gene therapy research STAR Grant winners announced - Pacific Biosciences - February 13th, 2024
- Lifetime Cost of DEB Gene Therapy Could Be $15-$22 Million - Medscape - February 13th, 2024
- 5 Things Youll Learn in Frederick Community Colleges New Cell and Gene Therapy Essentials Certificate ... - BioBuzz - February 13th, 2024
- Regenxbios investigational gene therapy shows promise in Hunter syndrome - PMLiVE - February 13th, 2024
- Topical Gene Therapy Effective in Treating Debilitating Eye Condition - InventUM - University of Miami - February 13th, 2024
- Bayer Says First Patient Randomized In Askbio Phase II Gene Therapy Trial - XM - February 13th, 2024
- Top 3 grants in regenerative medicine: January 2024 - RegMedNet - February 13th, 2024
- Realistic Expectations, Limitations With Gene Therapy for Parkinson Disease: Ignacio Mata, PhD - Neurology Live - February 5th, 2024
- Gene-Based Therapy May Slow Development of Life-Threatening Heart Condition - NYU Langone Health - February 5th, 2024
- After Promising Early Efficacy, Eli Lilly Eager to Study Hearing Loss Gene Therapy in More Children - precisionmedicineonline.com - February 5th, 2024
- Gene therapy hailed as medical magic wand for hereditary swelling disorder - The Guardian - February 5th, 2024
- France Approves Gene Therapy Clinical Trial to Treat Hearing Loss in Children - The Hearing Review - February 5th, 2024
- Early Trial Success for Inherited Deafness Gene-Therapy - RegMedNet - February 5th, 2024
- Can gene therapy be the key to restoring hearing loss? - Labiotech.eu - February 5th, 2024
- Jaguar Gene Therapy Announces FDA Clearance of IND to Study JAG201 in a Genetic Form of Autism Spectrum ... - Business Wire - February 5th, 2024
- FDA inspection clears Abeona's gene therapy BLA By Investing.com - Investing.com - February 5th, 2024
- Hearing Restored in Deaf Children in Gene Therapy Clinical Trial - Securities.io - February 5th, 2024
- Experimental gene therapy allows kids with inherited deafness to hear - The Associated Press - January 28th, 2024
- Children with genetic deafness have hearing restored with gene therapy: Study - ABC News - January 28th, 2024
- Positive Phase 1/2 Clinical Trial Data for an Investigational Gene Therapy for Genetic Hearing Loss to be Presented at ... - Investors | Eli Lilly and... - January 28th, 2024
- UChicago Medicine among the first in the country to offer newly approved sickle cell gene therapies - UChicago Medicine - January 28th, 2024
- Gene Therapy Allows an 11-Year-Old Boy to Hear for the First Time - The New York Times - January 28th, 2024
- Gene therapy restores hearing in children with hereditary deafness - EurekAlert - January 28th, 2024
- Experimental gene therapy helps treat kids with inherited hearing loss - CBS News - January 28th, 2024
- Children's Hospital of Philadelphia Performs First in U.S. Gene Therapy Procedure to Treat Genetic Hearing Loss - PR Newswire - January 28th, 2024
- Gene Therapy Brings Hearing to Kids With Congenital Deafness - U.S. News & World Report - January 28th, 2024
- Gene Therapy Restores Hearing in Small Trial for Kids With Hereditary Deafness - Technology Networks - January 28th, 2024
- Cell Therapies on Ice: Seven Trends in Cryopreservation To Enable Cell and Gene Therapy Research - Technology Networks - January 28th, 2024
- Gene Therapy Brings Hearing to Kids With Congenital Deafness - HealthDay - January 28th, 2024
- Gene therapies restore hearing in several kids with inherited deafness - Livescience.com - January 28th, 2024
- 11-Year-Old Boy Hears For First Time Ever Thanks To Gene Therapy Breakthrough - IFLScience - January 28th, 2024
- Innovative Gene Therapy Restores Hearing In Deaf Child, Offering Potential Cure - Study Finds - January 28th, 2024
- Sound of Success, Gene Therapy Breakthrough Grants Hearing to Deaf Children in China-Harvard Study - Hoodline - January 28th, 2024
- Perspectives in FTD Research Webinar: Gene Therapy for FTD -- What Do I Need to Know? | AFTD - AFTD - January 20th, 2024
- UK leads the way in cell and gene therapy - Research Professional News - January 20th, 2024
- Charles River's (CRL) New Launch to Boost Gene Therapy Programs - Yahoo Finance - January 20th, 2024
- UK maintains status as an attractive destination for advanced therapy clinical trials - BioPharma-Reporter.com - January 20th, 2024
- Understanding the impact of AI on the advancement of gene therapy development - pharmaphorum - January 20th, 2024
- Gene therapy TSHA-102 delivered to 1st child in REVEAL trial - Rett Syndrome News - January 20th, 2024
- AAV Vectors in Gene Therapy Market is Predicted to Observe Skyrocketed Growth During the Study Period (2019-2032 ... - PR Newswire - January 20th, 2024
- Family pins hopes on gene therapy - The Recorder and Times - January 20th, 2024
- Arbor, 4DMT partner on new gene therapies in ALS, other diseases - ALS News Today - January 11th, 2024
- The year of gene therapy? FDA's Marks calls for solutions to regulatory, manufacturing challenges - BioWorld Online - January 11th, 2024
- Gene Therapy Approvals Expected to Ramp Up in 2024 Amid Manufacturing, Cost Challenges - BioSpace - January 11th, 2024
- Turning On Gene Therapy with the pA Regulator - RegMedNet - January 11th, 2024
- Revolutionizing Healthcare: Gene Therapy Market Projected to Reach USD 46.5 billion by 2030 at a 22. - PharmiWeb.com - January 11th, 2024
- Regeneron CEO Reveals the Game-Changing Potential of Gene Therapy in Biotech! - BioTecNika - January 11th, 2024
- Bayer's AskBio checks off another early win for gene therapy platform with Parkinson's safety data - FierceBiotech - January 11th, 2024
- Are Rare Brain Diseases the Next Commercial Frontier for Gene Therapy? - MedCity News - January 3rd, 2024
- Novartis, Voyager Ink Up-to-$1.3B Expansion of Neuro Gene Therapy Collaboration - Genetic Engineering & Biotechnology News - January 3rd, 2024
- Novel switch turns genes on/off on cue, a promising step toward safer gene therapy - Phys.org - January 3rd, 2024
- Which Therapeutic Areas Experts Think Will Gain Traction In 2024 - Clinical Leader - January 3rd, 2024
- Voyager Therapeutics and Novartis Announce Major Licensing Deal in Gene Therapy Field - Medriva - January 3rd, 2024
- We Are About to Enter the Golden Age of Gene Therapy - Inverse - January 3rd, 2024
- Revolutionizing Gene Therapy: Baylor Researchers Develop RNA-Based Switch for Therapeutic Protein Control - BNN Breaking - January 3rd, 2024
- Casgevy and Lyfgenia: Two Gene Therapies Approved for Sickle Cell Disease - Yale Medicine - December 25th, 2023
- Top story: Family connects with researchers behind Canavan gene therapy - UMass Medical School - December 25th, 2023
- Excision Bio Seeks to Suppress HIV Replication With CRISPR Gene Therapy - AJMC.com Managed Markets Network - December 25th, 2023
- Hemgenix Gene Therapy Shows Long-Term Efficacy, Safety in Hemophilia B Patients - Managed Healthcare Executive - December 25th, 2023
- The missing piece of the puzzle - EUobserver - December 25th, 2023
- Brace yourselves for these trends in clinical research in 2024 - Labiotech.eu - December 25th, 2023
- RegMedNet's 2023 remix: top content of the year - RegMedNet - December 25th, 2023
- BU Researchers Helped Develop the First FDA-Approved Gene Therapies to Treat Sickle Cell Disease - Boston University - December 16th, 2023
- Research discovers that mice possess a natural gene therapy system - Phys.org - December 16th, 2023
- Cell- and gene-therapy hub bound for Lake Success; New York to invest up to $150 million - Newsday - December 16th, 2023