- Oral Presentation on In Vivo Gene Therapy Product Candidate and Data on Gene Editing Candidate Paved Way for Initiation of juMPStart and pheEDIT Trials -
BEDFORD, Mass., Oct. 20, 2021 (GLOBE NEWSWIRE) -- Homology Medicines, Inc. (Nasdaq: FIXX), a genetic medicines company, announced today presentations of nonclinical data and patient and caregiver feedback that support the Companys clinical programs for mucopolysaccharidosis type II (MPS II), or Hunter syndrome, and phenylketonuria (PKU). During the American Society of Human Genetics (ASHG) 2021 Virtual Meeting, an oral presentation included data from Homologys IND-enabling studies with HMI-203, a one-time, in vivo investigational gene therapy in development for the treatment of MPS II in the recently initiated Phase 1 juMPStart clinical trial. Patient and caregiver feedback on unmet medical needs in MPS II that informed trial design were also presented. Additionally, Homology shared results from a nonclinical study that demonstrated the precision of its nuclease-free, homologous recombination-based, in vivo gene editing candidate for PKU as the Company starts its first gene editing trial.
Our presentations at ASHG show the holistic approach we have taken to prepare for our Hunter syndrome gene therapy and PKU gene editing trials, demonstrating our commitment to thoroughly evaluating the product candidates and the needs of the patient community ahead of clinical studies, stated Albert Seymour, Ph.D., Chief Scientific Officer of Homology Medicines. Armed with patient and caregiver feedback on the unmet medical needs that persist, we recently initiated our juMPStart trial to evaluate a single-dose of HMI-203 in adults with Hunter syndrome. Additionally, integration assays from our in vivo PKU gene editing program demonstrated no evidence of off-target integration in human hepatocytes in a xenograft murine model and support our Phase 1 trial.
In the oral presentation titled, Long-Term Systemic Expression and Cross-Correction Ability of HMI-203: Investigational Gene Therapy Candidate for Mucopolysaccharidosis Type II or Hunter Syndrome, data showed that a single I.V. dose of HMI-203 in the MPS II murine model resulted in the following, through 52 weeks (end of study):
Story continues
Systemic expression of vector genomes, transcripts and functional I2S enzyme;
Secretion of active I2S into circulation demonstrating cross-correction;
Systemic reduction of disease-relevant biomarkers, including positive and significant correlation between cerebral glycosaminoglycan heparin sulfate (GAG-HS) and LAMP1 levels as well as cerebrospinal fluid (CSF) and cerebral GAG-HS levels; and
Prevention of progression of craniofacial and hindlimb deformities.
Also related to HMI-203, Patient-Focused Drug Development for a Single Intravenous Dose of HMI-203 Gene Therapy in Adult Mucopolysaccharidosis (MPS) II, or Hunter Syndrome, Patients included qualitative data on unmet medical needs from enzyme replacement therapy (ERT)-treated adult MPS II patients and/or their caregivers. They reported:
Weekly ERT infusions, surgeries and supportive therapies inadequately address range of motion and mobility, pain, and hearing loss;
Burdens associated with ERT and other therapies, including frequency and duration of treatment, and painful and extended recoveries;
High degree of anxiety regarding prognosis, longevity, need for more invasive surgeries, and financial challenges; and
Expectations for a potential one-time gene therapy include ability to maintain current quality of life with ERT independence.
To support HMI-103, its gene editing candidate for PKU, Homology also presented, Genome-Wide Integration Assay For rAAV Mediated Homologous Recombination (HR) in Human Hepatocytes Demonstrated Precision of In Vivo Gene Editing Approach. Using quantitative molecular methods, including long-read sequencing, in a murine liver model populated with human hepatocytes, a single I.V. dose of HMI-103 demonstrated on-target integration into the desired locus and no evidence of integration into any other genomic location.
For more information, please visit http://www.homologymedicines.com/publications.
About Homology Medicines, Inc. Homology Medicines, Inc. is a clinical-stage genetic medicines company dedicated to transforming the lives of patients suffering from rare diseases by addressing the underlying cause of the disease. The Companys lead clinical program, HMI-102, is an investigational gene therapy for adults with phenylketonuria (PKU) and additional programs focus on lysosomal storage disorders including Hunter syndrome, paroxysmal nocturnal hemoglobinuria (PNH) and other diseases. Homologys proprietary platform is designed to utilize its family of 15 human hematopoietic stem cell-derived adeno-associated virus vectors (AAVHSCs) to precisely and efficiently deliver genetic medicines in vivo through a gene therapy or nuclease-free gene editing modality, as well as to deliver one-time gene therapy to produce antibodies throughout the body through the GTx-mAb platform. Homology has a management team with a successful track record of discovering, developing and commercializing therapeutics with a focus on rare diseases. Homology believes its initial clinical data and compelling preclinical data, scientific and product development expertise, internal manufacturing capabilities and broad intellectual property position the Company as a leader in genetic medicines. For more information, visit http://www.homologymedicines.com.
Forward-Looking StatementsThis press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including without limitation statements regarding our expectations surrounding the potential, safety, efficacy, and regulatory and clinical progress of our product candidates; the potential of our gene therapy and gene editing platforms, including our GTx-mAb platform; our plans and timing for the release of additional preclinical and clinical data; our beliefs regarding our manufacturing capabilities; our position as a leader in the development of genetic medicines; and our participation in upcoming presentations and conferences. These statements are neither promises nor guarantees, but involve known and unknown risks, uncertainties and other important factors that may cause our actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements, including, but not limited to, the following: the impact of the COVID-19 pandemic on our business and operations, including our preclinical studies and clinical trials, and on general economic conditions; we have and expect to continue to incur significant losses; our need for additional funding, which may not be available; failure to identify additional product candidates and develop or commercialize marketable products; the early stage of our development efforts; potential unforeseen events during clinical trials could cause delays or other adverse consequences; risks relating to the capabilities of our manufacturing facility; risks relating to the regulatory approval process; interim, topline and preliminary data may change as more patient data become available, and are subject to audit and verification procedures that could result in material changes in the final data; our product candidates may cause serious adverse side effects; inability to maintain our collaborations, or the failure of these collaborations; our reliance on third parties; failure to obtain U.S. or international marketing approval; ongoing regulatory obligations; effects of significant competition; unfavorable pricing regulations, third-party reimbursement practices or healthcare reform initiatives; product liability lawsuits; failure to attract, retain and motivate qualified personnel; the possibility of system failures or security breaches; risks relating to intellectual property and significant costs as a result of operating as a public company. These and other important factors discussed under the caption Risk Factors in our Quarterly Report on Form 10-Q for the quarter ended June 30, 2021 and our other filings with the SEC could cause actual results to differ materially from those indicated by the forward-looking statements made in this press release. Any such forward-looking statements represent managements estimates as of the date of this press release. While we may elect to update such forward-looking statements at some point in the future, we disclaim any obligation to do so, even if subsequent events cause our views to change.
Company ContactsTheresa McNeelyChief Communications Officer and Patient Advocatetmcneely@homologymedicines.com781-301-7277
Media Contact:Cara Mayfield Vice President, Patient Advocacy and Corporate Communications cmayfield@homologymedicines.com 781-691-3510
Read the rest here:
Homology Medicines Announces Presentation of Data Supporting Clinical Programs in MPS II and PKU, including Nonclinical and Patient-Focused Research,...
- Morning Medical Update: Gut bacteria and longevity; People with ... - Medical Economics - June 12th, 2023 [June 12th, 2023]
- Artificial Intelligence and the Aging Process: A Match Made for ... - Down to Game - June 12th, 2023 [June 12th, 2023]
- Saint Bernard Lifespan: How Long Do Saint Bernards Live? - AZ Animals - June 12th, 2023 [June 12th, 2023]
- 'Human Resources' Was Poised to Be Netflix's Longest-Running ... - PRIMETIMER - June 12th, 2023 [June 12th, 2023]
- Early Life Adversity Doesn't Affect Gorillas Like Other Species - The Good Men Project - June 12th, 2023 [June 12th, 2023]
- Wellness luminaries, experts to meet in KK - The Borneo Post - June 12th, 2023 [June 12th, 2023]
- I grew up with hazardous smoke from forest fires in Asia. Here's what ... - Morningstar - June 12th, 2023 [June 12th, 2023]
- Workplace Networks Are More Important than Promotions to Engage ... - GlobeNewswire - June 12th, 2023 [June 12th, 2023]
- Accenture staff working at Google training AI move to unionise - The Australian Financial Review - June 12th, 2023 [June 12th, 2023]
- Hair Wigs and Extension Market Size to Grow by USD 5.26 billion from 2021 to 2026, Growth Driven by Technologi - Black Enterprise - April 10th, 2023 [April 10th, 2023]
- Want to age like a fine wine? Here are some tips for longevity and feeling younger - The Indian Express - February 18th, 2023 [February 18th, 2023]
- Human body | Organs, Systems, Structure, Diagram, & Facts - January 27th, 2023 [January 27th, 2023]
- Human | Avatar Wiki | Fandom - January 27th, 2023 [January 27th, 2023]
- Calorie Restriction and Fasting Diets: What Do We Know? - January 4th, 2023 [January 4th, 2023]
- Emma Thompson On Longevity Of Holiday Fave Love Actually But It Is SEX Actually That Is Earning Her Awards Buzz In Leo Grande - Deadline - December 26th, 2022 [December 26th, 2022]
- Heads or tails: What statistical models tell us about the probability of living beyond 110 - The Conversation - December 12th, 2022 [December 12th, 2022]
- LinkedIn founder and VC Reid Hoffman says human amplification via A.I. will revolutionize large enterprises in the next five years - Fortune - December 12th, 2022 [December 12th, 2022]
- Human hibernation is possible and could boost longevity | New Scientist - December 2nd, 2022 [December 2nd, 2022]
- Home - Human Longevity Institute - October 23rd, 2022 [October 23rd, 2022]
- Exercise: 7 benefits of regular physical activity - Mayo Clinic - October 23rd, 2022 [October 23rd, 2022]
- Life expectancy - Wikipedia - October 13th, 2022 [October 13th, 2022]
- Life Expectancy - Our World in Data - October 13th, 2022 [October 13th, 2022]
- The Longevity Investors Conference Gstaad brought together longevity experts and deep-pocketed investors - Cointelegraph - October 13th, 2022 [October 13th, 2022]
- The Search for a Pill That Can Help Dogsand HumansLive Longer - WIRED - October 13th, 2022 [October 13th, 2022]
- Aging of the Heart Correlates With a Poor Gut Microbiome - Lifespan.io News - October 13th, 2022 [October 13th, 2022]
- Aditxt, Inc. (Nasdaq: ADTX) Announces Development and Publication of a Mathematical Model for Predicting Longevity and Variations of Immune Response... - October 13th, 2022 [October 13th, 2022]
- FOXO Technologies Announces First Distribution Partner and Begins Product Rollout in California; Expands Executive Team With Insurance Industry Talent... - October 13th, 2022 [October 13th, 2022]
- A conversation with Holocaust scholar Dr. Irving Berkowitz - South Florida Sun Sentinel - October 13th, 2022 [October 13th, 2022]
- Nine postdoctoral fellowships in aging research awarded by the Glenn Foundation for Medical Research and AFAR - EurekAlert - October 13th, 2022 [October 13th, 2022]
- The 5 Wellness Habits That Slow Down Aging, Science Reveals Eat This Not That - Eat This, Not That - October 13th, 2022 [October 13th, 2022]
- Winning Federal Contracts on the Top 20 Contract Vehicles - Bloomberg Government - October 13th, 2022 [October 13th, 2022]
- Lawrence Austin promoted to rank of colonel - Winona Times - October 13th, 2022 [October 13th, 2022]
- The longevity dividend: Work in an era of 100-year lives - October 2nd, 2022 [October 2nd, 2022]
- Retirement Savings Calculator - Northwestern Mutual - October 2nd, 2022 [October 2nd, 2022]
- Clues to Human Longevity Unearthed in Largescale Mouse Genetic Study - Genetic Engineering & Biotechnology News - October 2nd, 2022 [October 2nd, 2022]
- Can we find ways to live beyond 100? Millionaires are betting on it. - MIT Technology Review - October 2nd, 2022 [October 2nd, 2022]
- Restrict Calories to Boost Immune Function - The Epoch Times - October 2nd, 2022 [October 2nd, 2022]
- The Black Progress Index: Examining the social factors that influence Black well-being - Brookings Institution - October 2nd, 2022 [October 2nd, 2022]
- Kevin Williamson on Scream 6 and Franchise Longevity - Bleeding Cool News - October 2nd, 2022 [October 2nd, 2022]
- 507-Year-Old Clam May Be the World's Oldest Living Being - History of Yesterday - October 2nd, 2022 [October 2nd, 2022]
- I should look forward to early retirement, but I think I'm dreading it - CBC.ca - October 2nd, 2022 [October 2nd, 2022]
- International Coffee Day 2022: How The Beloved Brew Helps Fight Cancer, Kidney Disease And Depression - ABP Live - October 2nd, 2022 [October 2nd, 2022]
- Corey Feldman And Jamison Newlander Talk The Lost Boys And The Birth Of The Two Coreys - Forbes - October 2nd, 2022 [October 2nd, 2022]
- Proven Levels of Human Longevity | NextBigFuture.com - September 11th, 2022 [September 11th, 2022]
- Prolonging healthspan by delaying ageing -- NUHS opens Singapore's first Centre for Healthy Longevity to increase healthy lifespan of Singapore... - September 11th, 2022 [September 11th, 2022]
- What is the Secret to Longevity in Real Estate? - CandysDirt.com - Candy's Dirt - September 11th, 2022 [September 11th, 2022]
- Jeff Stukey & Jay Olshansky, Ph.D with Wealthspan Advisors, Interviewed on the Influential Entrepreneurs Podcast Discussing How Aging Science... - September 11th, 2022 [September 11th, 2022]
- The Queen of the World - The Atlantic - September 11th, 2022 [September 11th, 2022]
- THR Icon: At 100, Norman Lear Looks Back (And Ahead) at Whats Changed Since the Maude Abortion Episode - Hollywood Reporter - September 11th, 2022 [September 11th, 2022]
- TIFF 2022: The Colour of Ink Review - ThatShelf.com - September 11th, 2022 [September 11th, 2022]
- Should Age Be on the Ballot? - Next Avenue - September 11th, 2022 [September 11th, 2022]
- Was The Spring Water At Balmoral Castle The Secret To Elizabeth II's Longevity? - Nation World News - September 11th, 2022 [September 11th, 2022]
- A complete guide to the health benefits of nuts europeantimes.news - The European Times - September 11th, 2022 [September 11th, 2022]
- Debut Launches its Bio2Consumer Platform to Become the Leader in Vertically Integrated Synthetic Biology - PR Web - September 11th, 2022 [September 11th, 2022]
- Rapamycin Drug Used in Cancer Therapy Found to Increase Human Lifespan [Study] - Nature World News - August 30th, 2022 [August 30th, 2022]
- Eating Healthy: Why Eating Grapes Would Increase Lifespan | The Guardian Nigeria News - Nigeria and World News Guardian Life The Guardian Nigeria... - August 30th, 2022 [August 30th, 2022]
- Beauty Bets on Longevity WWD - WWD - August 30th, 2022 [August 30th, 2022]
- Analysis of the impact of success on three dimensions of sustainability in 173 countries | Scientific Reports - Nature.com - August 30th, 2022 [August 30th, 2022]
- You might have glowing skin in no time by making few easy changes to your skincare routine - TDPel Media - August 30th, 2022 [August 30th, 2022]
- Nucleai Appoints New Head of Pathology to Support Expansion in Biopharmaceutical and Clinical Markets - Business Wire - August 8th, 2022 [August 8th, 2022]
- Daily AI Roundup: Biggest Machine Learning, Robotic And Automation Updates - AiThority - August 8th, 2022 [August 8th, 2022]
- Prolonged and intense heatwave affecting parts of western and northern Europe breaks temperature records; globally, July 2022 was one of three warmest... - August 8th, 2022 [August 8th, 2022]
- YLEM: Augmenting sustainable futures through material exploration and testing - STIRworld - August 8th, 2022 [August 8th, 2022]
- Do you want to live forever? Big Tech and the quest for eternal youth - The New Statesman - August 6th, 2022 [August 6th, 2022]
- This startup wants to copy you into an embryo for organ harvesting - MIT Technology Review - August 6th, 2022 [August 6th, 2022]
- Renewal Bio Acquires Breakthrough Stem Cell Technology With Applications in Infertility and Longevity - Benzinga - August 6th, 2022 [August 6th, 2022]
- Get married or get a degree to live longer says study, debunking theories of womens longevity over men - Times Now - August 6th, 2022 [August 6th, 2022]
- Nucleai Appoints New Head of Pathology to Support Expansion in Biopharmaceutical and Clinical Markets - StreetInsider.com - August 6th, 2022 [August 6th, 2022]
- Some species are immortal but dying of old age is humanity's secret weapon - The Telegraph - August 6th, 2022 [August 6th, 2022]
- Opinion: Changing When and How Much We Eat May Extend Health Span - The Scientist - August 6th, 2022 [August 6th, 2022]
- Vieroots uses genome testing to win over a growing tribe of wellness enthusiasts - YourStory - August 6th, 2022 [August 6th, 2022]
- Herbs radiate and give you youthful skin - The Hans India - August 6th, 2022 [August 6th, 2022]
- How Pop Culture, Politics, Science, and Business Got So Old - The Atlantic - August 6th, 2022 [August 6th, 2022]
- John Hood: How to read this column | Columnists | journalnow.com - Winston-Salem Journal - August 6th, 2022 [August 6th, 2022]
- How Long Is The Human Lifespan? Here's What Studies Have Found | mindbodygreen - mindbodygreen - July 31st, 2022 [July 31st, 2022]
- Employees are livingand workinglonger. CEOs are introducing initiatives like phased retirements to harness the longevity economy - Fortune - July 31st, 2022 [July 31st, 2022]
- Why Do We Get Old, and Can Aging Be Reversed? - Quanta Magazine - July 31st, 2022 [July 31st, 2022]
- The Secret to Longevity? Here Are 7 Health Tech Gadgets That Can Help - The Recursive - July 31st, 2022 [July 31st, 2022]
- Four Fitness Facts to Fuel Your Workout - The New York Times - July 31st, 2022 [July 31st, 2022]
- Chris Reed: Has the Fountain of Youth been found just west of Interstate 5? - The San Diego Union-Tribune - July 31st, 2022 [July 31st, 2022]