ASPA

WAKEFIELD, Mass., Nov. 7, 2023 /PRNewswire/ -- Myrtelle Inc. ("Myrtelle" or the "Company"), a clinical-stage gene therapy company focused on developing transformative treatments for neurodegenerative diseases, today announced that Christopher Janson, M.D., Principal Investigator on Myrtelle's Canavan Disease Gene Therapy Trial and Associate Professor of Neuroscience & Founding Director of Human Gene Therapy Center at Premier Health Wright State Neuroscience Institute in Dayton, Ohio, will deliver a presentation entitled "Translation of Gene Therapy Brain Biomarkers from Nonclinical Science to Clinical Trials: A Case Study in Canavan Disease" on Tuesday, November 7, 2023, at the 5th Annual Gene Therapy for Neurological Disorders Summit in Boston, Massachusetts. Dr. Janson's presentation will highlight encouraging data to date from Myrtelle's Phase 1/2 gene therapy clinical study of the Company's proprietary rAAV vector in patients with Canavan disease (CD). The rAAV vector directly targets oligodendrocytes, the brain cells affected in CD that are responsible for producing myelin – the insulating material that enables proper neuronal function. In CD, normal brain development is impaired due to a mutation in the ASPA gene that encodes the enzyme aspartoacylase. The lack of normal aspartoacylase activity negatively impacts myelin production and brain development. This unique oligodendrocyte-targeting rAAV vector is a novel gene therapy approach directed at restoring ASPA function and brain development specifically in these target cells in patients with CD. The presentation will review encouraging post-treatment improvements through the primary analysis timepoint at one year in the Phase 1/2 clinical study, including emerging data on biomarkers identified by magnetic resonance imaging (MRI) that precede clinical score improvements in motor and cognitive function using validated scales. Specifically, statistically significant early gains in target white matter and myelin were observed following gene therapy which predict later improvements in specific functional scores on the validated Gross Motor Function Measure and Mullen Scales of Early Learning. Improvements generally continued to progress from the 6-month timepoint to the 12-month readout. As presented previously, treated patients were also observed to outperform an untreated age-matched natural history cohort on multiple measures. Dr. Janson's presentation will also cover foundational nonclinical data illustrating the gene therapy efficacy in model systems. "Biomarker improvements observed on MRI imaging in patients are supportive of underlying recovery of brain anatomy following gene therapy treatment. These results reinforce the hypothesis that addressing the gene deficit directly in the brain, and specifically oligodendrocytes, of patients with Canavan disease can potentially restore myelination and support functional improvements," said Dr. Janson. "We look forward to sharing details at the upcoming summit." Date/Time: Tuesday, November 7, 2023, 2:30 pm Location: Hilton Back Bay Hotel, Boston, MA ABOUT MYRTELLE Myrtelle Inc. is a gene therapy company focused on developing transformative treatments for neurodegenerative diseases. The company has a proprietary platform, intellectual property, and portfolio of programs and technologies supporting innovative gene therapy approaches for neurodegenerative diseases. Myrtelle has an exclusive worldwide licensing agreement with Pfizer Inc. for its Canavan disease program. For more information, please visit the Company's website at: . ABOUT CANAVAN DISEASE Canavan disease (CD) is a fatal childhood genetic brain disease caused by mutations in the ASPA gene (ASPA) which prevent the normal expression of aspartoacylase, a critical enzyme produced in oligodendrocytes. The lack of normal aspartoacylase expression negatively impacts brain development, including myelin production. Patients with CD are impacted at birth but may appear normal until several months old when symptoms begin to develop. Poor head control, abnormally large head size, difficulty in eye tracking, excessive irritability, severely diminished muscle tone, and delays in reaching motor milestones, such as rolling, sitting, and walking, are the typical initial manifestations of CD. As the disease progresses, seizures, spasticity, difficulties in swallowing, and overall muscle deterioration emerge with most affected children developing life-threatening complications by approximately 10 years of age. Currently, there are no cures for CD, and only palliative treatments are available. More information on Myrtelle's clinical trial in Canavan disease can be found on under the identifier NCT04833907 or by emailing PatientAdvocacy@MyrtelleGTX.com. Forward-Looking Statements This press release contains forward-looking statements. Words such as "may," "believe," "will," "expect," "plan," "anticipate," "estimate," "intend" and similar expressions (as well as other words or expressions referencing future events, conditions, or circumstances) are intended to identify forward-looking statements. Forward-looking statements are based upon current estimates and assumptions and include statements regarding results obtained to date continuing to reinforce the hypothesis that addressing the gene deficit directly in the brain, and specifically in oligodendrocytes, of patients with Canavan disease can potentially restore myelination and support functional improvements. While Myrtelle believes these forward-looking statements are reasonable, undue reliance should not be placed on any such forward-looking statements, which are based on information available to us on the date of this release. These forward-looking statements are subject to various risks and uncertainties, many of which are difficult to predict, that could cause actual results to differ materially from current expectations and assumptions from those set forth or implied by any forward-looking statements. Important factors that could cause actual results to differ materially from current expectations include, among others, Myrtelle's program demonstrating safety and efficacy, as well as future results that are consistent with prior results, the ability to generate the data needed for further development of this novel gene therapy in the patients with CD, and the ability to continue its clinical trials and to complete them on time and achieve the desired results. All forward-looking statements are based on Myrtelle's expectations and assumptions as of the date of this press release. Actual results may differ materially from these forward-looking statements. Except as required by law, Myrtelle expressly disclaims any responsibility to update any forward-looking statement contained herein, whether as a result of new information, future events, or otherwise.

WAKEFIELD, Mass.--(BUSINESS WIRE)-- Myrtelle Inc. (“Myrtelle” or the “Company”), a gene therapy company focused on developing transformative treatments for neurodegenerative diseases, today announced that updated positive data from the one-year readout in the Company’s open-label Phase 1/2 First-in-Human (FIH) clinical trial for Canavan disease (CD) using the Company’s recombinant adeno-associated virus (rAAV) vector-based investigational gene therapy will be presented by Mark Hurtt, M.D., Acting Chief Medical Officer, in a talk entitled “MYR-101 Gene Therapy for Canavan Disease - Preliminary One-Year Results” on Wednesday, October 11, 2023, at the upcoming Cell & Gene Meeting on the Mesa in Carlsbad, CA. Myrtelle’s FIH trial utilizes the Company’s proprietary rAAV vector to directly target oligodendrocytes, the brain cells affected in CD that are responsible for producing myelin – the insulating material that enables proper neuronal function. In CD, normal brain development is impaired due to a mutation in the ASPA gene that encodes the enzyme aspartoacylase. The lack of normal aspartoacylase activity negatively impacts myelin production and brain development. The Company’s unique oligodendrocyte-targeting rAAV vector is the only gene therapy approach known to us directed at restoring ASPA function and brain development specifically in these target cells in patients with CD. The presentation will review encouraging post-treatment improvements through the primary analysis timepoint of one year in the Phase 1/2 clinical study for multiple functional and anatomic measurements, including clinical improvements in motor and cognitive function using validated scales. Following gene therapy, statistically significant gains in certain target white matter and myelin were observed. In turn, improvements in multiple domains on the validated Gross Motor Function Measure and Mullen Scales of Early Learning were demonstrated. Improvements generally continued to progress from the 6-month timepoint to the 12-month readout. Treated patients were routinely observed to outperform untreated age-matched natural history patients on the Mullen Scale of Early Learning. “Results obtained to date are encouraging and continue to reinforce the hypothesis that addressing the ASPA genetic deficit specifically in oligodendrocytes in patients with Canavan disease can potentially restore oligodendrocyte health and myelination and support functional improvements,” said Dr. Hurtt. “We look forward to sharing details at the upcoming presentation and to discussing further development with global regulatory authorities in support of potential approvals.” Organized by the Alliance for Regenerative Medicine, the Cell & Gene Meeting on the Mesa is a three-day conference featuring more than 120 dedicated company presentations by leading public and private companies, highlighting technical and clinical achievements over the past 12 months in the areas of cell therapy, gene therapy, gene editing, tissue engineering, and broader regenerative medicine technologies, as well as over 100 panelists and featured speakers. Date/Time: Wednesday, October 11, 2023, 9:45 am Location: Ecolab Life Sciences Ballroom – Park Hyatt Aviara Resort 7100 Resort Dr., Carlsbad, CA ABOUT MYRTELLE Myrtelle Inc. is a gene therapy company focused on developing transformative treatments for neurodegenerative diseases. The company has a proprietary platform, intellectual property, and portfolio of programs and technologies supporting innovative gene therapy approaches for neurodegenerative diseases. Myrtelle has an exclusive worldwide licensing agreement with Pfizer Inc. for its Canavan disease program. For more information, please visit the Company’s website at: . ABOUT CANAVAN DISEASE Canavan disease (CD) is a fatal childhood genetic brain disease caused by mutations in the ASPA gene (ASPA) which prevent the normal expression of aspartoacylase, a critical enzyme produced in oligodendrocytes. The lack of normal aspartoacylase expression negatively impacts brain development, including myelin production. Patients with CD are impacted at birth but may appear normal until several months old when symptoms begin to develop. Poor head control, abnormally large head size, difficulty in eye tracking, excessive irritability, severely diminished muscle tone, and delays in reaching motor milestones, such as rolling, sitting, and walking, are the typical initial manifestations of CD. As the disease progresses, seizures, spasticity, difficulties in swallowing, and overall muscle deterioration emerge with most affected children developing life-threatening complications by approximately 10 years of age. Currently, there are no cures for CD, and only palliative treatments are available. More information on Myrtelle’s clinical trial in Canavan disease can be found on under the identifier NCT04833907 or by emailing PatientAdvocacy@MyrtelleGTX.com. Forward-Looking Statements This press release contains forward-looking statements. Words such as “may,” “believe,” “will,” “expect,” “plan,” “anticipate,” “estimate,” “intend” and similar expressions (as well as other words or expressions referencing future events, conditions, or circumstances) are intended to identify forward-looking statements. Forward-looking statements are based upon current estimates and assumptions and include statements regarding results obtained to date continuing to reinforce the hypothesis that addressing the ASPA genetic deficit specifically in oligodendrocytes in patients with Canavan disease can potentially restore oligodendrocyte health and myelination and support functional improvements and potential regulatory approvals. While Myrtelle believes these forward-looking statements are reasonable, undue reliance should not be placed on any such forward-looking statements, which are based on information available to us on the date of this release. These forward-looking statements are subject to various risks and uncertainties, many of which are difficult to predict, that could cause actual results to differ materially from current expectations and assumptions from those set forth or implied by any forward-looking statements. Important factors that could cause actual results to differ materially from current expectations include, among others, Myrtelle’s program demonstrating safety and efficacy, as well as future results that are consistent with prior results, the ability to generate the data needed for further development of this novel gene therapy in the patients with CD, and the ability to continue its clinical trials and to complete them on time and achieve the desired results. All forward-looking statements are based on Myrtelle’s expectations and assumptions as of the date of this press release. Actual results may differ materially from these forward-looking statements. Except as required by law, Myrtelle expressly disclaims any responsibility to update any forward-looking statement contained herein, whether as a result of new information, future events, or otherwise.

Resilience to manufacture BridgeBio’s lead investigational gene therapy treatments, BBP-631 and BBP-812 Partnership provides an innovative, capital efficient, sustainable model for BridgeBio to develop, test and deliver transformative medicines for patients more quickly PALO ALTO, Calif. and SAN DIEGO, Oct. 03, 2023 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. (BridgeBio), a commercial-stage biopharmaceutical company focused on genetic diseases and cancers, and National Resilience, Inc. (Resilience), a technology-focused biomanufacturing company dedicated to broadening access to complex medicines, today announced a strategic collaboration to manufacture and advance BBP-812, an investigational adeno-associated virus (AAV) 9 gene therapy for Canavan disease, and BBP-631, an investigational AAV 5 gene therapy for congenital adrenal hyperplasia (CAH). The two companies have developed a novel manufacturing and aligned incentive business model to drive these gene therapies forward with an emphasis on sustainability and capital-efficiency. Under the terms of the collaboration, BridgeBio will transfer its manufacturing process for its lead AAV-based gene therapy candidates to Resilience’s network of gene therapy sites. As part of an innovative cost and risk-sharing framework, Resilience will provide in-kind manufacturing services and will receive future development and approval milestones and low-to-mid single digit royalties on BBP-631 and BBP-812. Resilience will support the ongoing clinical development manufacturing needs and will serve as the primary commercial manufacturer for both programs if successful. “Our partnership with BridgeBio seeks to accelerate development of innovative therapeutic options for patients in need,” said Rahul Singhvi, Sc.D., Chief Executive Officer of Resilience. “We are pleased to partner with a gene therapy and rare disease leader, and we are inspired by their passion to deliver medicines to patients.” Beyond BBP-812 and BBP-631, Resilience will also be the primary manufacturer for future clinical projects across BridgeBio’s gene therapy portfolio. The agreement will reduce manufacturing uncertainty for these programs and is expected to help BridgeBio expedite development of gene therapies going forward. “Manufacturing is the most critical and costly aspect of developing gene therapy for patients with a serious unmet need. We conduct process development, analytical development and optimization in our own labs, and with this partnership we can now hand these programs off to one of the most trusted partners in the industry for scale up and commercial manufacturing. This allows us to accelerate the development of our gene therapy portfolio in a capital-efficient and sustainable way with the hope of providing medicines more quickly,” said Eric David, M.D., J.D., chief executive officer of BridgeBio Gene Therapy. “We appreciate the opportunity to collaborate with the experienced and knowledgeable team at Resilience on the manufacturing of our gene therapies. We hope this expedites our path to benefiting as many patients as possible, as soon as possible,” said Neil Kumar, Ph.D., founder and CEO of BridgeBio. About BridgeBio Pharma, Inc. BridgeBio Pharma, Inc. is a commercial-stage biopharmaceutical company founded to discover, create, test, and deliver transformative medicines to treat patients who suffer from genetic diseases and cancers with clear genetic drivers. BridgeBio’s pipeline of development programs ranges from early science to advanced clinical trials. BridgeBio was founded in 2015 and its team of experienced drug discoverers, developers and innovators are committed to applying advances in genetic medicine to help patients as quickly as possible. For more information visit bridgebio.com and follow us on LinkedIn and Twitter. About BBP-812 BBP-812 is an investigational AAV9 gene therapy for Canavan disease. Using AAV gene therapy, BridgeBio seeks to deliver functional copies of the ASPA gene throughout the body and into the brain, potentially correcting the disease at its source. Preclinical proof-of-concept results have shown the approach restores survival and normal motor function in Canavan disease models. BBP-812 was granted Fast Track Designation, Rare Pediatric Drug Designation, and Orphan Drug Designation by the U.S. Food and Drug Administration. BBP-812 was also granted Orphan Drug Designation by the European Medicines Agency. About BBP-631 BBP-631 is an AAV5 gene therapy developed to treat CAH due to 21-hydroxylase deficiency at its source. BBP-631 is designed to deliver a functional copy of the 21-hydroxylase gene and has been shown through multiple preclinical studies to result in efficient and persistent delivery to the adrenal gland, where hormones are naturally made. If successful, BBP-631 may restore the body’s hormone and steroid balance by enabling people with CAH to naturally make their own cortisol and aldosterone. It could also allow for people with CAH to eliminate or significantly reduce their daily glucocorticoid or mineralocorticoid doses, which is the current standard of care for patients. About Resilience Resilience is a technology-focused biomanufacturing company dedicated to broadening access to complex medicines. Founded in 2020, the company is building a sustainable network of high-tech, end-to-end manufacturing solutions to ensure the treatments of today and tomorrow can be made quickly, safely, and at scale. By continuously advancing the science of biopharmaceutical manufacturing and development, Resilience seeks to free its partners to focus on the discoveries that improve patients’ lives and protect biopharmaceutical supply chains against future disruptions. For more information, visit and follow us on social media: @IncResilience on Twitter and Resilience on LinkedIn. BridgeBio Pharma, Inc. Forward-Looking Statements This press release contains forward-looking statements. Statements BridgeBio makes in this press release may include statements that are not historical facts and are considered forward-looking within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, Section 27A of the Securities Act of 1933, as amended (the Securities Act), and Section 21E of the Securities Exchange Act of 1934, as amended (the Exchange Act), which are usually identified by the use of words such as “anticipates,” “believes,” “estimates,” “expects,” “intends,” “may,” “plans,” “projects,” “seeks,” “should,” “will,” and variations of such words or similar expressions. BridgeBio intends these forward-looking statements to be covered by the safe harbor provisions for forward-looking statements contained in Section 27A of the Securities Act and Section 21E of the Exchange Act. These forward-looking statements, including statements relating to BridgeBio’s collaboration agreement with Resilience and the milestone and royalty payments thereunder, the ability of BridgeBio and Resilience’s model to drive BridgeBio’s gene therapies forward in a capital-efficient and sustainable way, the success and impact of the collaboration with Resilience, including on reducing manufacturing uncertainty and expediting development of BridgeBio’s gene therapy portfolio; and the potential of our related gene therapy programs, if successful, to benefit patients with CAH or Canavan disease, reflect our current views about our plans, intentions, expectations and strategies, which are based on the information currently available to us and on assumptions BridgeBio has made. Although BridgeBio believes that its plans, intentions, expectations, strategies and prospects as reflected in or suggested by those forward-looking statements are reasonable, BridgeBio can give no assurance that the plans, intentions, expectations or strategies will be attained or achieved. Furthermore, actual results may differ materially from those described in the forward-looking statements and will be affected by a number of risks, uncertainties and assumptions, including, but not limited to, BridgeBio’s ability to continue and complete its ongoing and planned clinical trials of BBP-631 for the treatment of CAH disease and of BBP-812 for the treatment of Canavan disease, initial and ongoing data from clinical trials not being indicative of final data; the design and success of BridgeBio’s ongoing and planned clinical trials, difficulties with enrollment in BridgeBio’s clinical trials, adverse events that may be encountered in BridgeBio’s clinical trials, the FDA or other regulatory agencies not agreeing with BridgeBio’s regulatory approval strategies, components of BridgeBio’s filings, such as clinical trial designs, conduct and methodologies, or the sufficiency of data submitted, as well as those risks set forth in the Risk Factors section of BridgeBio’s Annual Report on Form 10-K for the year ended December 31, 2022 and its other filings with the U.S Securities and Exchange Commission. Moreover, BridgeBio operates in a very competitive and rapidly changing environment in which new risks emerge from time to time. These forward-looking statements are based upon the current expectations and beliefs of BridgeBio’s management as of the date of this press release, and are subject to certain risks and uncertainties that could cause actual results to differ materially from those described in the forward-looking statements. Except as required by applicable law, BridgeBio assumes no obligation to update publicly any forward-looking statements, whether as a result of new information, future events or otherwise. No representations or warranties (expressed or implied) are made about the accuracy of any such forward-looking statements. Media Contacts: BridgeBio Vikram Bali contact@bridgebio.com (650) 789-8220 Resilience Michele Roberts Head of Communications Michele.roberts@Resilience.com (888) 737-2460 Tatjana Heller Head of Investor Relations and Analytics Tatjana.heller@resilience.com (888) 737-2460