MECP2

Data from an analysis of five NHP studies showed lumbar IT administration led to widespread and consistent biodistribution of AAV9 gene therapy vectors across brain and spinal cord regions IT and ICM administration achieved comparable biodistribution across brain and spinal cord regions in NHPs Findings from the analysis reaffirm the clinical potential of IT administration as an effective, safe and minimally invasive approach to deliver AAV-based gene therapies designed to treat CNS diseases in both children and adults DALLAS, Oct. 22, 2024 (GLOBE NEWSWIRE) -- Taysha Gene Therapies, Inc. (Nasdaq: TSHA) (Taysha or the Company), a clinical-stage biotechnology company focused on advancing adeno-associated virus (AAV)-based gene therapies for severe monogenic diseases of the central nervous system (CNS), today announced that it will present biodistribution data from an analysis of 28 non-human primates (NHP) evaluating the delivery of AAV9 gene therapy vectors across five different studies using intrathecal (IT) delivery by lumbar puncture (four studies) or intra-cisterna magna (ICM) injection (one study). The data will be presented during a poster presentation at the upcoming 31st Annual Congress of the European Society of Gene & Cell Therapy (ESGCT), taking place in Rome, Italy from October 22-25, 2024. These findings reinforce Taysha’s clinical development approach utilizing IT administration to deliver AAV-based gene therapies designed to treat the genetic root cause of CNS diseases, including the TSHA-102 program in clinical evaluation for children, adolescents and adults living with Rett syndrome. “Findings from an analysis across five NHP studies showed that both IT and ICM administration led to comparable, consistent and widespread biodistribution of AAV9 vector throughout the brain and spinal cord regions,” said Sukumar Nagendran, M.D., President and Head of Research & Development at Taysha. “Importantly, these findings further support the clinical potential of IT administration as an effective, safe and minimally invasive delivery approach for broad targeting of the CNS that has potential for outpatient use in both children and adults. We believe the NHP biodistribution data, together with our additional preclinical data, reaffirm our clinical development strategy utilizing IT administration and provide translational support for the broad clinical effect reported following treatment with TSHA-102 in both our REVEAL adolescent/adult trial and REVEAL pediatric trial." Poster presentation details are as follows: Title: Broad CNS Biodistribution of AAV9-based Gene Therapies Delivered by Intrathecal Lumbar Puncture in Non-Human Primates Presenters: Nino Devidze, MS, Ph.D., Clinical Development Lead and Emdadul Haque, Ph.D., Senior Director, Translational Sciences at Taysha Gene Therapies Date and Time: Wednesday October 23, 2024, from 13:30 to 15:00 CEST Poster Session: CNS & Sensory Diseases Poster Number: P0284 Additional details on the meeting can be found at the 31st Annual ESGCT Congress website. About TSHA-102TSHA-102 is a self-complementary intrathecally delivered AAV9 investigational gene transfer therapy in clinical evaluation for Rett syndrome. Designed as a one-time treatment, TSHA-102 aims to address the genetic root cause of the disease by delivering a functional form of MECP2 to cells in the CNS. TSHA-102 utilizes a novel miRNA-Responsive Auto-Regulatory Element (miRARE) technology designed to mediate levels of MECP2 in the CNS on a cell-by-cell basis without risk of overexpression. TSHA-102 has received Regenerative Medicine Advanced Therapy, Fast Track and Orphan Drug and Rare Pediatric Disease designations from the FDA, Orphan Drug designation from the European Commission and Innovative Licensing and Access Pathway designation from the Medicines and Healthcare products Regulatory Agency. About Rett SyndromeRett syndrome is a rare neurodevelopmental disorder caused by mutations in the X-linked MECP2 gene encoding methyl CpG-binding protein 2 (MeCP2), which is essential for regulating neuronal and synaptic function in the brain. The disorder is characterized by loss of communication and hand function, slowing and/or regression of development, motor and respiratory impairment, seizures, intellectual disabilities and shortened life expectancy. Rett syndrome progression is divided into four key stages, beginning with early onset stagnation at 6 to 18 months of age followed by rapid regression, plateau and late motor deterioration. Rett syndrome primarily occurs in females and is one of the most common genetic causes of severe intellectual disability. Currently, there are no approved disease-modifying therapies that treat the genetic root cause of the disease. Rett syndrome caused by a pathogenic/likely pathogenic MECP2 mutation is estimated to affect between 15,000 and 20,000 patients in the U.S., EU, and U.K. About Taysha Gene TherapiesTaysha Gene Therapies (Nasdaq: TSHA) is a clinical-stage biotechnology company focused on advancing adeno-associated virus (AAV)-based gene therapies for severe monogenic diseases of the central nervous system. Its lead clinical program TSHA-102 is in development for Rett syndrome, a rare neurodevelopmental disorder with no approved disease-modifying therapies that address the genetic root cause of the disease. With a singular focus on developing transformative medicines, Taysha aims to address severe unmet medical needs and dramatically improve the lives of patients and their caregivers. The Company’s management team has proven experience in gene therapy development and commercialization. Taysha leverages this experience, its manufacturing process and a clinically and commercially proven AAV9 capsid in an effort to rapidly translate treatments from bench to bedside. For more information, please visit www.tayshagtx.com. Forward-Looking StatementsThis press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as “anticipates,” “believes,” “expects,” “intends,” “projects,” “plans,” and “future” or similar expressions are intended to identify forward-looking statements. Forward-looking statements include, but are not limited to, statements concerning the potential of TSHA-102 and Taysha’s other product candidates to positively impact quality of life and alter the course of disease in the patients Taysha seeks to treat, its research, development and regulatory plans for its product candidates, the potential for these product candidates to receive regulatory approval from the FDA or equivalent foreign regulatory agencies, and whether, if approved, these product candidates will be successfully distributed and marketed and the potential market opportunity for Taysha’s product candidates. Forward-looking statements are based on management’s current expectations and are subject to various risks and uncertainties that could cause actual results to differ materially and adversely from those expressed or implied by such forward-looking statements. Accordingly, these forward-looking statements do not constitute guarantees of future performance, and you are cautioned not to place undue reliance on these forward-looking statements. Risks regarding Taysha’s business are described in detail in its SEC filings, including in Taysha’s Annual Report on Form 10-K for the full-year ended December 31, 2023 and Quarterly Report on Form 10-Q for the quarter ended June 30, 2024, which are available on the SEC’s website at www.sec.gov. Additional information will be made available in other filings that Taysha makes from time to time with the SEC. These forward-looking statements speak only as of the date hereof, and Taysha disclaims any obligation to update these statements except as may be required by law. Company Contact:Hayleigh Collins Director, Head of Corporate Communications and Investor RelationsTaysha Gene Therapies, Inc.hcollins@tayshagtx.com Media Contact:Carolyn HawleyInizio EvokeCarolyn.hawley@inizioevoke.com

FARMINGDALE, N.Y., Oct. 02, 2024 (GLOBE NEWSWIRE) -- DepYmed, Inc. (“DepYmed” or the “Company”) is a clinical-stage pharmaceutical company focused on developing transformative medicines for many poorly treated diseases that may benefit significantly by targeting the protein tyrosine phosphatase (PTP) family of enzymes. Today, DepYmed announces co-founder and research advisor Nicholas Tonks, Ph.D. will present data at the 9th World Rett Syndrome Congress in Queensland, Australia being held October 2-5, 2024. Dr. Tonks’ presentation, titled, “PTP1B inhibition – a clinic-ready, mechanism-based approach to the treatment of Rett syndrome” will occur on October 3, 2024 at 4:30PM local time. In the presentation, Dr. Tonks discusses the nature of Rett syndrome as an X-linked neurological disorder caused primarily by mutations in a transcriptional regulator, methyl CpG binding protein 2 (MECP2). He then outlines the preclinical work completed for the Company’s PTP1B inhibitor, DPM-1003, which improved a wide range of Rett syndrome phenotypes in RTT mice. He concludes that these data provide validation of PTP1B as a mechanism-based, therapeutic target for Rett syndrome, suggesting DPM-1003 as a new approach to therapeutic intervention in the disease. DPM-1003 is currently poised to enter clinical development and has been cleared by the FDA for clinical testing in the United States under an IND. At this time, the Company is actively raising funds to support this clinical initiative. The 9th World Rett Syndrome Congress includes the latest in research, clinical innovations, and drugs. Seminars will be presented by world-renowned experts in Rett syndrome. The Conference is hosted by the Rett Syndrome Association of Australia, a not-for-profit organization managed by a committee of volunteers most of whom are parents of Rett syndrome children. The Association seeks to enhance the quality of life for persons with the syndrome and their families, and to be of assistance to anyone interested in the disorder. About DPM-1003 Protein tyrosine phosphatases are major players in the control of cell signaling pathways that are disrupted in many diseases, yet to date no drug modulators of these enzymes have been successfully developed. DepYmed is the first company to develop a new class of orally bioavailable drug candidates that act by allosterically inhibiting PTP1B, one of the most important PTP drug targets. One of its lead compounds has shown promising efficacy in preclinical models of Rett syndrome, and the Company hopes to initiate a phase 1 clinical trial in 2024. DPM-1003 has been granted Orphan Drug designation by the U.S. Food and Drug Administration and conditionally designated under the FDA’s Rare Pediatric Disease program. About Rett Syndrome Rett syndrome is a progressive neurodevelopmental disorder that almost exclusively affects females. Infants with Rett syndrome generally develop normally for 6 to 18 months after birth. At this point, they lose previously acquired skills (developmental regression) such as purposeful hand movements and the ability to communicate. Affected children often develop autistic-like behaviors, breathing irregularities, feeding and swallowing difficulties, growth retardation, and seizures. Most Rett syndrome cases are caused by mutations of the MECP2 gene on the X chromosome and can present with a wide range of disability ranging from mild to severe. The expression of PTP1B, DepYmed’s target in Rett syndrome, is regulated by MECP2; in turn, PTP1B controls important cell functions that are disrupted in the disease. About DepYmed, Inc. DepYmed Inc., is a New York based rare disease and cancer therapeutic development company that was founded to capitalize on the scientific discoveries of the Tonks lab at Cold Spring Harbor Laboratory in the physiological function of PTP1B and ways to modulate its role in various human diseases. DepYmed is currently developing a new class of potent, orally bioavailable small molecule allosteric inhibitors of the enzyme PTP1B as novel therapeutics for Rett Syndrome, different types of cancer, metabolic diseases, obesity, and Alzheimer’s Disease where the PTP1B target has shown promise. In addition, DepYmed has also discovered a novel class of small molecules with copper chelating properties that it is developing as potential therapeutic agents for diseases such as Wilson disease and various cancers. The company is actively developing a deep pipeline of new compounds with broad therapeutic potential in these emerging drug classes in collaboration with Cold Spring Harbor Laboratory. For more information, please visit the Company’s website at: www.depymed.com. For Media Inquiries please contact: Jules AbrahamJQA Partners, Inc.jabraham@jqapartners.com 917-885-7378 DepYmed:info@depymedinc.com

DALLAS, Sept. 26, 2024 (GLOBE NEWSWIRE) -- Taysha Gene Therapies, Inc. (Nasdaq: TSHA) (Taysha or the Company), a clinical-stage biotechnology company focused on advancing adeno-associated virus (AAV)-based gene therapies for severe monogenic diseases of the central nervous system (CNS), today announced that it will present previously disclosed clinical data on its TSHA-102 program in evaluation for Rett syndrome during an oral presentation at the upcoming 9th World Rett Syndrome Congress, taking place in Queensland, Australia from October 2-5, 2024. The presentation will entail previously disclosed positive clinical data up to 52 weeks from the ongoing REVEAL Phase 1/2 adolescent and adult trial and initial clinical data up to 22 weeks from the REVEAL Phase 1/2 pediatric trial evaluating TSHA-102 in Rett syndrome. The Company expects to provide clinical data from cohort two (high dose, n=3) and a longer-term update on clinical data from cohort one (low dose, n=2) from both the adolescent and adult, and the pediatric REVEAL trials in the first half of 2025. Oral presentation details are as follows: Abstract Title: First cohort data from the REVEAL adolescent/adult and pediatric studies of TSHA-102 gene therapy for Rett syndrome Presenter: Sukumar Nagendran, M.D., President and Head of Research and Development at Taysha Gene Therapies Session: Preclinical and Clinical Stream Date & Time: Friday, October 4, 2024, from 12:40-1:00 PM Australian Eastern Standard Time (AEST) Additional details on the meeting can be found at the 9th World Rett Syndrome Congress website. About TSHA-102TSHA-102 is a self-complementary intrathecally delivered AAV9 investigational gene transfer therapy in clinical evaluation for Rett syndrome. Designed as a one-time treatment, TSHA-102 aims to address the genetic root cause of the disease by delivering a functional form of MECP2 to cells in the CNS. TSHA-102 utilizes a novel miRNA-Responsive Auto-Regulatory Element (miRARE) technology designed to mediate levels of MECP2 in the CNS on a cell-by-cell basis without risk of overexpression. TSHA-102 has received Regenerative Medicine Advanced Therapy, Fast Track and Orphan Drug and Rare Pediatric Disease designations from the FDA, Orphan Drug designation from the European Commission and Innovative Licensing and Access Pathway designation from the Medicines and Healthcare products Regulatory Agency. About Rett SyndromeRett syndrome is a rare neurodevelopmental disorder caused by mutations in the X-linked MECP2 gene encoding methyl CpG-binding protein 2 (MeCP2), which is essential for regulating neuronal and synaptic function in the brain. The disorder is characterized by loss of communication and hand function, slowing and/or regression of development, motor and respiratory impairment, seizures, intellectual disabilities and shortened life expectancy. Rett syndrome progression is divided into four key stages, beginning with early onset stagnation at 6 to 18 months of age followed by rapid regression, plateau and late motor deterioration. Rett syndrome primarily occurs in females and is one of the most common genetic causes of severe intellectual disability. Currently, there are no approved disease-modifying therapies that treat the genetic root cause of the disease. Rett syndrome caused by a pathogenic/likely pathogenic MECP2 mutation is estimated to affect between 15,000 and 20,000 patients in the U.S., EU, and U.K. About Taysha Gene TherapiesTaysha Gene Therapies (Nasdaq: TSHA) is a clinical-stage biotechnology company focused on advancing adeno-associated virus (AAV)-based gene therapies for severe monogenic diseases of the central nervous system. Its lead clinical program TSHA-102 is in development for Rett syndrome, a rare neurodevelopmental disorder with no approved disease-modifying therapies that address the genetic root cause of the disease. With a singular focus on developing transformative medicines, Taysha aims to address severe unmet medical needs and dramatically improve the lives of patients and their caregivers. The Company’s management team has proven experience in gene therapy development and commercialization. Taysha leverages this experience, its manufacturing process and a clinically and commercially proven AAV9 capsid in an effort to rapidly translate treatments from bench to bedside. For more information, please visit www.tayshagtx.com. Forward-Looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as “anticipates,” “believes,” “expects,” “intends,” “projects,” “plans,” and “future” or similar expressions are intended to identify forward-looking statements. Forward-looking statements include, but are not limited to, statements concerning the potential of TSHA-102 and Taysha’s other product candidates, to positively impact quality of life and alter the course of disease in the patients Taysha seeks to treat, its research, development and regulatory plans for its product candidates, including the anticipated timelines for reporting data for the TSHA-102 REVEAL trials and the trial design of the TSHA-102 REVEAL trials, the potential for these product candidates to receive regulatory approval from the FDA or equivalent foreign regulatory agencies, and whether, if approved, these product candidates will be successfully distributed and marketed and the potential market opportunity for Taysha’s product candidates. Forward-looking statements are based on management’s current expectations and are subject to various risks and uncertainties that could cause actual results to differ materially and adversely from those expressed or implied by such forward-looking statements. Accordingly, these forward-looking statements do not constitute guarantees of future performance, and you are cautioned not to place undue reliance on these forward-looking statements. Risks regarding Taysha’s business are described in detail in its SEC filings, including in Taysha’s Annual Report on Form 10-K for the full-year ended December 31, 2023 and Quarterly Report on Form 10-Q for the quarter ended June 30, 2024, which are available on the SEC’s website at www.sec.gov. Additional information will be made available in other filings that Taysha makes from time to time with the SEC. These forward-looking statements speak only as of the date hereof, and Taysha disclaims any obligation to update these statements except as may be required by law. Company Contact:Hayleigh Collins Director, Head of Corporate Communications and Investor RelationsTaysha Gene Therapies, Inc.hcollins@tayshagtx.com Media Contact:Carolyn HawleyInizio EvokeCarolyn.hawley@inizioevoke.com