CHU Sainte-Justine

MONTREAL, Sept. 26, 2024 (GLOBE NEWSWIRE) -- From September 28 to October 1, the Shriners Hospitals for Children - Canada will host the 4th International Symposium on Arthrogryposis. Organized in collaboration with the other Montreal pediatric centers - CHU Sainte-Justine, Centre de réadaptation Marie Enfant and Montreal Children's Hospital - the symposium will take place at the Hôtel Omni Mont-Royal, 1050 Sherbrooke Street West. Pre-conference workshops will be held on Saturday, September 28 at the Shriners Hospitals for Children - Canada. Arthrogryposis is a group of rare diseases that occur in around 1 in 3,000 children. Arthrogryposis manifests itself as joint stiffness, present from birth and affecting all four limbs to varying degrees. It may be accompanied by other signs: skeletal, visceral or neurological. People with arthrogryposis live with a limited range of movement, often restricting their participation in daily activities. Arthrogryposis is one of a wide range of so-called fetal immobility syndromes, encompassing a multitude of neurological and muscular abnormalities whose inventory is not yet complete. Over the course of four days, experts from around the world will discuss ways of approaching and treating this pathology, as well as the latest research projects in progress. For Dr. Reggie Hamdy and Dr. Noémi Dahan-Oliel, co-chairs of the scientific and organizing committees, the need for this 4th international symposium is clear. Noémi Dahan-Oliel, clinical researcher and associate professor at McGill University, explains: “By promoting knowledge sharing and networking among an international community of experts and people living with this condition, we are on the lookout for scientific advances and discoveries that will enable optimal evidence-based care. One of our Quebec stars, Luca “Lazylegz” Patuelli, www.lucapatuelli.com, a member of the organizing committee for this 4th edition of the international symposium, will perform a dance show on Sunday, September 29 at the Omni Hotel's Salle Pierre de Coubertin during the cocktail reception at 5:45 pm. Luca embodies the “can-do attitude” of people living with a rare disease. He is proof that support for patients and families, as called for by Quebec's policy on rare diseases, is not only an inescapable necessity, but also a source of wealth.” “The Shriners Healthcare Network is at the forefront of clinical advancements, and its Genomics Institute seeks to better understand the genetic causes of rare diseases such as arthrogryposis. It's by working together with clinicians, researchers and people living with arthrogryposis that we can learn, innovate and move forward to meet the needs of this community,” says Reggie Hamdy, MD, MSc (Ortho) FRCSC, orthopaedic surgeon and director of the Arthrogryposis Clinic at the Shriners in Montréal. Several patients and families living with arthrogryposis will also be there speaking about the care they received and their life experiences. To view the symposium program and find out more about the guest speakers: https://event.fourwaves.com/fr/amcinternationalsymposium/pages The first international symposium on arthrogryposis was held in 2007 in Birmingham, UK, to discuss different aspects of care for people with arthrogryposis. These learnings set the tone for the second symposium, held in St Petersburg, Russia, in 2014. Clinical and research advances made in the field of arthrogryposis were shared at the 3rd symposium in Philadelphia, USA, in 2018. The hospital, in collaboration with other Montreal pediatric facilities, is proud to welcome the 4th International Arthrogryposis Symposium to Montreal! About Shriners Hospitals for Children – Canada Established in Montreal in 1925, Shriners Hospital for Children ─ Canada is the only Canadian facility within the extensive Shriners Hospitals network. A bilingual hospital, it offers ultra-specialized acute orthopedic care to children from Canada, the United States and around the world. The hospital's mission is to promote health and provide treatment and rehabilitation for infants, children and young adults with orthopedic and neuromuscular conditions such as scoliosis, osteogenesis imperfecta, clubfoot, hip dysplasia and cerebral palsy. The hospital is committed to excellence and innovation in clinical practice, research and education. Affiliated with McGill University, the hospital enables resident physicians and healthcare professionals to take advantage of its facilities on the Glen site to further their training and experience. In addition to offering a patient- and family-centered care environment, the hospital is present in communities across Canada through telemedicine, outreach clinics and satellite clinics. Information: Emmanuelle RondeauCommunicationsShriners Hospitals for Children – CanadaTel.: 514-282-6990; Cell.: 514-207-1057erondeau@shrinenet.org A photo accompanying this announcement is available at https://www.globenewswire.com/NewsRoom/AttachmentNg/7dc067b6-e47b-49a5-99fd-8507debbcdf2

Company to host webcast on Tuesday, June 18, 2024, at 8:00 AM Eastern Time Oral presentation by Elsa Rossignol, M.D., FRCP, FAAP, Principal Investigator of the REVEAL adolescent and adult trial at CHU Sainte-Justine and Colleen Buhrfiend, M.D., of RUSH University Medical Center at the 2024 IRSF Rett Syndrome Scientific Meeting on Wednesday, June 19, 2024, at 11:00 AM Mountain Time DALLAS, June 12, 2024 (GLOBE NEWSWIRE) -- Taysha Gene Therapies, Inc. (Nasdaq: TSHA), 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 host a webcast on Tuesday, June 18, 2024, to discuss new clinical data from the REVEAL Phase 1/2 adolescent and adult trial and the REVEAL Phase 1/2 pediatric trial evaluating TSHA-102 in Rett syndrome, as well as provide a program update. The data will also be presented at the 2024 International Rett Syndrome Foundation (IRSF) Rett Syndrome Scientific Meeting taking place in Westminster, Colo., June 18-19, 2024. Taysha-Hosted Webcast DetailsDate/Time: Tuesday, June 18, 2024, at 8:00 AM Eastern TimeRegistration: https://lifescievents.com/event/taysha-2/The live webcast and replay will be available through Taysha’s website here. 2024 IRSF Rett Syndrome Scientific Meeting Presentation DetailsPoster PresentationTitle: TSHA-102 gene therapy for Rett syndrome: First-cohort data from the REVEAL Adolescent/Adult and Pediatric studies Date/Time: Tuesday, June 18, 2024, 5:15-7:00 PM Mountain TimePresenters: Elsa Rossignol, M.D., FRCP, FAAP, Associate Professor in Neuroscience and Pediatrics at the Université de Montréal and Principal Investigator of the REVEAL adolescent and adult trial at CHU Sainte-Justine and Colleen Buhrfiend, M.D., Assistant Professor of Pediatrics at RUSH University Medical Center Oral PresentationTitle: TSHA-102 gene therapy for Rett syndrome: First-cohort data from the REVEAL Adolescent/Adult and Pediatric studiesDate/Time: Wednesday, June 19, 2024, at 11:00 AM Mountain Time Presenters: Elsa Rossignol, M.D., FRCP, FAAP, Associate Professor in Neuroscience and Pediatrics at the Université de Montréal and Principal Investigator of the REVEAL adolescent and adult trial at CHU Sainte-Justine and Colleen Buhrfiend, M.D., Assistant Professor of Pediatrics at RUSH University Medical Center About Taysha Gene Therapies Taysha 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. 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

Data from first adult patient in REVEAL Phase 1/2 trial showed TSHA-102 was well-tolerated with no treatment-emergent SAEs as of 20-week assessment with sustained improvement across key efficacy measures and new improvement in R-MBA, PGI-I and hand function, a hallmark characteristic of Rett syndrome at week 12 Data from second adult patient showed TSHA-102 was well-tolerated with no treatment-emergent SAEs as of six-week assessment with improvement across key efficacy measures, including CGI-I, R-MBA, PGI-I and RSBQ at week four Notable differences in genetic mutation and phenotypic expression reported between patient one and two; Principal Investigator (PI) observed improvements in both patients across multiple domains, including autonomic function, socialization, and gross and fine motor skills, including further improvement in ability to sit unassisted at week 12 in patient one and improved posture, gait and stability at week four in patient two IDMC provided clearance to dose third adult patient based on available data; dosing of third adult patient and completion of cohort one (low dose) expected in the fourth quarter of 2023/first quarter of 2024; dosing of first pediatric patient in the U.S. expected in the first quarter of 2024 Entered into loan and security agreement with Trinity Capital that extends cash runway into 2026 and includes no financial covenants or warrants Conference call and live webcast today at 4:30 PM Eastern Time DALLAS, Nov. 14, 2023 (GLOBE NEWSWIRE) -- Taysha Gene Therapies, Inc. (Nasdaq: TSHA) (“Taysha” or “the Company”), a clinical-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS), today reported financial results for the third quarter ended September 30, 2023, and provided corporate and clinical updates. “Prior to initiating the REVEAL trial, the expectation of seeing a clinical benefit in adults with stage four Rett syndrome was low due to the advanced and relentless progression of the disease. We are highly encouraged by the positive 12-week data from the first adult patient and initial four-week data from the second adult patient in the low dose TSHA-102 cohort,” said Sean P. Nolan, Chairman and Chief Executive Officer of Taysha. “Importantly, response was seen across multiple clinical domains in both stage four patients with different genetic mutation severity and phenotypic expression, including autonomic function, socialization, and gross and fine motor skills. These early improvements in both patients, coupled with the sustained response through week 12 in the first patient, support the transformative potential of TSHA-102 across multiple genotypes of Rett syndrome.” Dr. Elsa Rossignol, M.D., FRCP, FAAP, Associate Professor in Neuroscience and Pediatrics at the Université de Montréal, and Principal Investigator of the REVEAL trial at the CHU Sainte-Justine added, “The two adult patients dosed with TSHA-102 have different mutations in their MECP2 gene that manifest in different phenotypes and clinical severity. Following treatment, both patients experienced improvement in key clinical domains impacting activities of daily living, including breathing dysrhythmia, autonomic function, socialization, and gross and fine motor skills. Both patients display significantly reduced breathing dysrhythmia, with less breath holding spells and infrequent hyperventilation, improved limb perfusion and vastly improved interest in social communication and activities. In addition, the first patient experienced sustained and new improvements, with restored movement in her legs and the gained ability to sit unassisted for up to 15 minutes for the first time in over a decade. Further, her hand function improved with the gained ability to grasp objects with her non-dominant hand and transfer them to her dominant hand for the first time since infancy. Following treatment, the second patient’s posture, gait and stability improved, resulting in straighter posture and smoother movements when walking. Her hand stereotypies also improved for the first time since regression at age three: she now displays less forceful hand wringing and her hands are often open and relaxed, providing new opportunities for fine motor skill learning. In addition, her seizures are much less frequent. I’m encouraged by the early positive signals and consistent improvement seen in both patients following treatment.” Recent Corporate Highlights Presented two posters at the European Society of Gene & Cell Therapy (ESGCT) 30th Annual Congress on new preclinical in vitro data supporting the miRARE technology, and initial clinical data from the first adult patient dosed in the REVEAL Phase 1/2 trialUnited States (U.S.) Food and Drug Administration (FDA) granted Fast Track Designation to TSHA-102 for Rett syndromeEntered into a loan and security agreement with Trinity Capital and terminated existing loan and security agreement with Silicon Valley Bank, extending cash runway into 2026; no financial covenants or warrants associated with the loan and security agreement with Trinity Capital Recent Clinical Highlights TSHA-102 in Rett syndrome: a self-complementary intrathecally delivered AAV9 gene transfer therapy in clinical evaluation for Rett syndrome, a rare genetic neurodevelopmental disorder caused by mutations in the X-linked MECP2 gene. TSHA-102 utilizes a novel miRARE technology designed to mediate levels of MECP2 in the CNS on a cell-by-cell basis without risk of overexpression. TSHA-102 is being evaluated in the REVEAL Phase 1/2 adult trial, a first-in-human, open-label, randomized, dose-escalation and dose-expansion study in Canada evaluating the safety and preliminary efficacy of TSHA-102 in adult females with Rett syndrome due to MECP2 loss-of-function mutation. Results from the first patient (large MECP2 deletion; associated with severe phenotype) and second patient (missense MECP2 mutation; associated with milder phenotype) with late motor deterioration stage four Rett syndrome dosed with TSHA-102 in the low dose cohort: Generally well-tolerated with no treatment-emergent serious adverse events (SAEs) as of 20-week assessment post-treatment for patient one and six-week assessment for patient twoBased on clinical observations by the Principal Investigator (PI), both patients demonstrated improvement in multiple clinical domains, with sustained and new improvements in patient one 12-weeks post-treatment and initial improvements in patient two four-weeks post-treatment, including: Autonomic function: improved breathing patterns and sleep quality/duration (patient one) reduced seizures and improved breathing patterns (patient two)Socialization: improved social interest and vocalization (patient one) improved social interest (patient two)Gross motor skills: gained ability to sit unassisted and move legs (patient one) improved posture, gait and stability (patient two)Fine motor skills: improved hand function (patient one) improved hand stereotypies (patient two) Seizure Diary demonstrated comparable seizure events relative to baseline through 20-weeks post-treatment in patient one and reduced seizure events relative to baseline through day 33 post-treatment for patient two, based on caregiver-reported medical historyClinical improvements demonstrated in both patients across key efficacy measures include: Patient one: sustained improvement through 12-weeks in Clinical Global Impression–Improvement (CGI-I), Clinical Global Impression–Severity (CGI-S) and Rett Syndrome Behavior Questionnaire (RSBQ), with new improvements in Revised Motor Behavior Assessment (R-MBA), Parental Global Impressions–Improvement (PGI-I) and Rett Syndrome Hand Function Scale (RSHFS)Patient two: improvement four-weeks post-treatment in CGI-I, PGI-I, RSBQ and R-MBA Figure accompanying this announcement is available at: https://www.globenewswire.com/NewsRoom/AttachmentNg/9b39103b-685c-4849-9072-97f32658320c. Additional information on available clinical data is available in the Company’s quarterly report on Form 10-Q for the quarter ended September 30, 2023, to be filed with the SEC.Independent Data Monitoring Committee (IDMC) provided clearance to dose third adult patient based on available data Upcoming Milestones Dosing of third adult patient and completion of dosing in cohort one (low dose) in the adult trial in Canada anticipated in the fourth quarter of 2023/first quarter of 2024Further updates on available clinical data from the low dose cohort expected in the first quarter of 2024Dosing of first pediatric Rett syndrome patient in the U.S. anticipated in the first quarter of 2024U.K. Medicines and Healthcare products Regulatory Agency (MHRA) response to Clinical Trial Application (CTA) for TSHA-102 in pediatric patients with Rett syndrome expected by year-end 2023 Third Quarter 2023 Financial Highlights Research and Development Expenses: Research and development expenses were $11.8 million for the three months ended September 30, 2023, compared to $16.8 million for the three months ended September 30, 2022. The net change was due to a $9.3 million decrease due to lower compensation expense as a result of reduced headcount, lower licensing milestone fees, fewer manufacturing batches and fewer raw material purchases. This was partially offset by a $4.3 million increase in activity surrounding ongoing clinical trial efforts in the Rett syndrome REVEAL adult and pediatric studies. General and Administrative (G&A) Expenses: General and administrative expenses were $8.6 million for the three months ended September 30, 2023, compared to $8.7 million for the three months ended September 30, 2022. The decrease of $0.1 million was due to reduced compensation expense due to lower headcount of $2.0 million and reduced consulting and professional fees of $0.7 million, partially offset by $2.6 million issuance costs allocated to the liability-classified pre-funded warrants issued in connection with the private placement financing completed in August 2023. Net loss: Net loss for the three months ended September 30, 2023, was $117.1 million, or $0.93 per share, as compared to a net loss of $26.5 million, or $0.65 per share, for the three months ended September 30, 2022, due to a non-cash expense of $100.5 million recorded in Q3 2023 from a change in the fair value of warrant liability from pre-funded warrants in connection with the private placement financing completed in August 2023. Cash and cash equivalents: As of September 30, 2023, the Company had cash and cash equivalents of $164.3 million. The Company expects that its existing cash and cash equivalents will fund operating expenses and capital requirements into 2026. Conference Call and Webcast Information Taysha management will hold a conference call and webcast today at 4:30 pm ET to review its financial and operating results and to provide corporate and clinical updates. The dial-in number for the conference call is 877-407-0792 (U.S./Canada) or 201-689-8263 (international). The conference ID for all callers is 13741244. The live webcast and replay may be accessed by visiting Taysha’s website at https://ir.tayshagtx.com/news-events/events-presentations. An archived version of the webcast will be available on the website for 30 days. About Taysha Gene Therapies Taysha Gene Therapies (Nasdaq: TSHA) is on a mission to eradicate monogenic CNS disease. With a singular focus on developing curative medicines, we aim to rapidly translate our treatments from bench to bedside. We have combined our team’s proven experience in gene therapy drug development and commercialization with the world-class UT Southwestern Gene Therapy Program. Together, we leverage our fully integrated platform with a goal of dramatically improving patients’ lives. More information is available at 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 statements concerning the potential of our product candidates, including the reproducibility and durability of any favorable results initially seen in our first and second patients dosed in the REVEAL trial and including our preclinical product candidates, to positively impact quality of life and alter the course of disease in the patients we seek to treat, our research, development and regulatory plans for our product candidates, the timing of our clinical trials, including reporting data therefrom, the forecast of our cash runway and the Company’s expectations regarding funding, operating and working capital expenditures. 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 our business are described in detail in our Securities and Exchange Commission (“SEC”) filings, including in our Annual Report on Form 10-K for the full-year ended December 31, 2022, and our Quarterly Report on Form 10-Q for the quarter ended September 30, 2023, both of which are available on the SEC’s website at www.sec.gov. Additional information will be made available in other filings that we make from time to time with the SEC. These forward-looking statements speak only as of the date hereof, and we disclaim any obligation to update these statements except as may be required by law. Taysha Gene Therapies, Inc.Condensed Consolidated Balance Sheet Data(in thousands, except share and per share data)(Unaudited) September 30,2023 December 31,2022ASSETS Current assets: Cash and cash equivalents $164,278 $87,880 Prepaid expenses and other current assets 5,529 8,537 Assets held for sale 2,000 — Total current assets 171,807 96,417 Restricted cash 2,637 2,637 Property, plant and equipment, net 11,169 14,963 Operating lease right-of-use assets 9,852 10,943 Other non-current assets 304 1,316 Total assets $ 195,769 $ 126,276 LIABILITIES AND STOCKHOLDERS' (DEFICIT) EQUITY Current liabilities: Accounts payable $7,520 $10,946 Accrued expenses and other current liabilities 13,638 18,287 Deferred revenue 18,759 33,557 Warrant liability 140,534 — Total current liabilities 180,451 62,790 Deferred revenue, net of current portion 2,951 — Term loan, net 38,548 37,967 Operating lease liability, net of current portion 19,101 20,440 Other non-current liabilities 3,832 4,130 Total liabilities 244,883 125,327 Stockholders' (deficit) equity Preferred stock, $0.00001 par value per share; 10,000,000 shares authorized and no shares issued and outstanding as of September 30, 2023 and December 31, 2022 — — Common stock, $0.00001 par value per share; 200,000,000 shares authorized and 186,960,193 and 63,207,507 issued and outstanding as of September 30, 2023 and December 31, 2022, respectively 2 1 Additional paid-in capital 511,632 402,389 Accumulated deficit (560,748) (401,441)Total stockholders’ (deficit) equity (49,114) 949 Total liabilities and stockholders' (deficit) equity $ 195,769 $ 126,276 Taysha Gene Therapies, Inc.Condensed Consolidated Statement of Operations(in thousands, except share and per share data)(Unaudited) For the Three MonthsEnded September 30, For the Nine MonthsEnded September 30, 2023 2022 2023 2022 Revenue $4,746 $— $11,847 $— Operating expenses: Research and development 11,791 16,774 44,096 78,462 General and administrative 8,589 8,683 23,328 30,019 Impairment of long-lived assets 616 — 616 — Total operating expenses 20,996 25,457 68,040 108,481 Loss from operations (16,250) (25,457) (56,193) (108,481)Other income (expense): Change in fair value of warrant liability (100,456) — (100,456) — Interest income 1,109 9 1,651 50 Interest expense (1,471) (1,078) (4,285) (2,493)Other expense (19) (1) (24) (12)Total other expense, net (100,837) (1,070) (103,114) (2,455)Net loss $ (117,087) $ (26,527) $ (159,307) $ (110,936)Net loss per common share, basic and diluted $ (0.93) $(0.65) $ (1.88) $ (2.79)Weighted average common shares outstanding, basic and diluted 125,700,799 40,937,808 84,630,796 39,761,764 Company Contact:Hayleigh Collins Director, Head of Corporate Communications and Investor RelationsTaysha Gene Therapies, Inc.hcollins@tayshagtx.com Media Contact:Carolyn HawleyCanale Communications carolyn.hawley@canalecomm.com