TSHA Taysha Gene Therapies Inc.

12.69
-0.69  -5%
Previous Close 13.38
Open 13.1
52 Week Low 11.46
52 Week High 33.35
Market Cap $488,234,362
Shares 38,473,945
Float 17,049,670
Enterprise Value $380,415,384
Volume 81,504
Av. Daily Volume 156,559
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Upcoming Catalysts

Drug Stage Catalyst Date
TSHA-120
Giant axonal neuropathy (GAN)
Phase 1/2
Phase 1/2
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AAV9/CLN7
CLN7 Disease
Phase 1
Phase 1
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TSHA-101
GM2 gangliosidosis
Phase 1/2
Phase 1/2
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TSHA-118
CLN1 Batten disease
Phase 1/2
Phase 1/2
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TSHA-102
Rett syndrome
Phase 1/2
Phase 1/2
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Drug Pipeline

Drug Stage Notes
TSHA-104
SURF1-associated Leigh syndrome
Phase 1/2
Phase 1/2
Phase 1/2 trial to be initiated by end of 2022.

Latest News

  1. Anticipate clinical safety and MFM32 functional data for TSHA-120 from the highest dose cohort of 3.5x1014 total vg in GAN in December 2021

    Expect preliminary clinical safety data and Hex A enzyme activity in the plasma and cerebral spinal fluid for TSHA-101 in GM2 gangliosidosis in December 2021

    Anticipate preliminary clinical data for the first-generation construct in CLN7 disease including safety data from first patient ever dosed intrathecally at 1.0x1015 total vg in December 2021

    On track to initiate Phase 1/2 trials for TSHA-118 in CLN1 disease and TSHA-102 in Rett syndrome by year-end 2021

    Completed five successful concurrent GMP campaigns for multiple programs to date, sufficient to support clinical-stage programs this year

    Received

    Anticipate clinical safety and MFM32 functional data for TSHA-120 from the highest dose cohort of 3.5x1014 total vg in GAN in December 2021

    Expect preliminary clinical safety data and Hex A enzyme activity in the plasma and cerebral spinal fluid for TSHA-101 in GM2 gangliosidosis in December 2021

    Anticipate preliminary clinical data for the first-generation construct in CLN7 disease including safety data from first patient ever dosed intrathecally at 1.0x1015 total vg in December 2021

    On track to initiate Phase 1/2 trials for TSHA-118 in CLN1 disease and TSHA-102 in Rett syndrome by year-end 2021

    Completed five successful concurrent GMP campaigns for multiple programs to date, sufficient to support clinical-stage programs this year

    Received Orphan Drug Designation from the European Commission for TSHA-101 for GM2 gangliosidosis, TSHA-102 for Rett syndrome and TSHA-105 for SLC13A5-related epilepsy

    Successfully completed nine regulatory meetings with multiple agencies to support clinical trial initiations; submitted initial request for end-of-Phase meeting to align regulatory pathway for TSHA-120 in giant axonal neuropathy (GAN)

    Recent publications of positive preclinical data from an AAV-mediated UBE3A gene replacement approach for Angelman syndrome and TSHA-104 for the treatment of SURF1-associated Leigh syndrome further support clinical development strategies

    Conference call and live webcast today at 8:00 AM Eastern Time

    Taysha Gene Therapies, Inc. (NASDAQ:TSHA), a patient-centric, pivotal-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) in both rare and large patient populations, today reported financial results for the third quarter ended September 30, 2021 and provided a corporate update.

    "We made significant advances in multiple aspects of our business, including nine productive regulatory meetings across several programs, a strategic addition of the clinical-stage CLN7 program and publication of positive preclinical data related to the Angelman syndrome and SURF1-associated Leigh syndrome programs," noted RA Session II, President, Founder and CEO of Taysha. "In September, we submitted an end-of-Phase meeting request for TSHA-120 with a major ex-US regulatory agency and look forward to submitting additional requests to multiple regulatory agencies by the end of this year. In GAN, we have up to 6 years of longitudinal data in individual patients and collectively 55-patient years of clinical safety and efficacy data from our ongoing clinical study. We also have eight years of robust longitudinal data from a natural history study being conducted at the NIH by Dr. Carsten Bönnemann. There has been consistency in the strength of data across multiple functional and biomarker endpoints, including the MFM32 scale, ophthalmological assessments and nerve biopsies. We look forward to reporting clinical safety and functional MFM32 data for TSHA-120 from the high dose cohort of 3.5x1014 total vg in December, where we believe continued clinically meaningful slowing of disease progression similar to that achieved with the lower dose cohorts would be considered confirmation of disease modification. For TSHA-101 in GM2 gangliosidosis, we remain on track to report preliminary clinical safety data and Hex A enzyme activity in plasma and CSF in December. Based on natural history, 2% to 4% Hex A enzyme activity in plasma normalizes survival and significantly improves clinical phenotype of GM2 gangliosidosis. For the CLN7 program, we anticipate preliminary clinical data including safety data for the first patient in history to be dosed at 1.0x1015 total vg with the first-generation construct in December. In parallel, we expect to finalize the second-generation CLN7 construct by year-end and initiate a planned pivotal clinical trial in 2022 with reference to clinical data from the first-generation construct. For TSHA-102 in Rett syndrome, we intend to submit an IND/CTA filing in November followed by initiation of a clinical trial by the end of this year. We have recently obtained preclinical data showing improvement in survival, and respiratory and motor functions in relevant animal models. Notably, preliminary data from a GLP toxicology study in non-human primates demonstrated no adverse findings at the highest dose tested suggesting that the miRARE platform is successfully downregulating MECP2 expression to within normal physiological levels. On the manufacturing front, we have completed five concurrent GMP campaigns to support multiple clinical programs this year," continued Mr. Session.

    "We believe the combination of our experience in gene therapy drug development, clinical pipeline and a strong balance sheet provides us with the financial and operational flexibility to achieve numerous value-generating milestones across our programs, and importantly, a potential regulatory approval for TSHA-120 in GAN in the near term. We look forward to continued execution on our development and regulatory strategies, and will provide updates throughout the remainder of this year," concluded Mr. Session.

    Recent Corporate Highlights

    • Publication of AAV-mediated UBE3A gene replacement approach for the treatment of Angelman syndrome in the Journal of Clinical Investigation Insight (JCI Insight)
    • Sponsored genetic testing for giant axonal neuropathy (GAN) in partnership with GeneDx, Inc to support inclusion of the genetic marker for GAN in the GeneDx hereditary neuropathy panel at no cost to individuals at risk for or suspected of having GAN
    • Announced collaboration with Hereditary Neuropathy Foundation and Charcot-Marie-Tooth Association Centers of Excellence to increase GAN disease awareness and access to testing
    • Obtained an exclusive option from UT Southwestern (UTSW) to license worldwide rights to a clinical-stage AAV9 gene therapy replacement program for the treatment of CLN7 disease
    • Entered into a research collaboration with UTSW to develop a next-generation construct for the treatment of CLN7 disease, which is expected to improve potency, safety profile, packaging efficiency and manufacturability over the first-generation construct
    • Provided a grant to Batten Hope, the leading CLN7 patient advocacy group, to support patient education, disease awareness and newborn screening initiatives
    • Announced publication of positive preclinical data for TSHA-104 demonstrating therapeutic potential in SURF-1-associated Leigh syndrome in Molecular Therapy: Methods & Clinical Development
    • Completed five successful concurrent GMP campaigns for multiple programs to date, sufficient to support clinical-stage programs this year. 187,000 square foot internal manufacturing facility on track to be fully operational in 2023
    • Received Orphan Drug Designation from the European Commission for TSHA-105 for SLC13A5-related epilepsy, TSHA-102 for Rett syndrome and TSHA-101 for GM2 gangliosidosis
    • Hosted CLN1 Investor Day on August 30th highlighting the company's TSHA-118 program
      • TSHA-118-treated CLN1 knockout mice demonstrated persistent supraphysiological levels of active PPT1, improved survival rates and sustained preservation of motor function
      • Insights from Key Opinion Leaders on preclinical data, utility of natural history data and current clinical trial design
    • Hosted Rett Syndrome Investor Day on September 22nd highlighting the company's TSHA-102 program
      • miRARE reduced overall expression of miniMeCP2 transgene expression and regulated genotype-dependent myc-tagged miniMECP2 expression across different brain regions on a cell-by-cell basis
      • Reviewed preclinical and natural history data that support an anticipated IND/CTA filing
    • Hosted Angelman Syndrome Investor Day on October 26th highlighting the company's two-pronged approach to treating Angelman syndrome
      • Two therapeutic approaches target entire Angelman syndrome population using gene replacement strategy on UBE3A to mimic the maternal UBE3A allele expression and a vectorized knockdown of UBE3A-ATS to unsilence the paternal allele
      • AAV-mediated UBE3A gene replacement recapitulates endogenous isoform ratios by replacing both the short and long isoforms of UBE3A in key regions of the brain, leading to improvements in motor learning, behavior outcomes, and seizure phenotypes in Angelman mouse models

    Anticipated Milestones by Program

    TSHA-120 for GAN: an intrathecally dosed AAV9 gene therapy currently being evaluated in a clinical trial for the treatment of GAN, a rare inherited genetic disorder that affects both the central and peripheral nervous systems and is caused by loss-of-function mutations in the gene coding for gigaxonin

    • Based on a longitudinal prospective natural history study conducted at the NIH by Dr. Carsten Bönnemann, there is a predictable decline in the MFM32 score of approximately 8-points per year across all patients regardless of age. A 4-point change in MFM32 is considered clinically meaningful. To date, we have up to eight years of robust data from this study.
    • Currently in the GAN program, we have up to 6 years of longitudinal data in individual patients and collectively 55-patient years of clinical safety and efficacy data from our ongoing clinical study with no drug-related serious adverse events, no signs of acute or subacute inflammation, no sudden sensory changes and no drug-related or persistent elevation of transaminases
    • In December, anticipate clinical safety and functional MFM32 data for TSHA-120 from the highest dose cohort of 3.5x1014 total vg in GAN, with continued clinically meaningful slowing of disease progression similar to that achieved with the lower dose cohorts would be considered confirmation of disease modification
    • Request for end-of-Phase meeting with ex-US regulatory agency for TSHA-120 completed in September with a preliminary meeting date in January 2022. Additional submissions expected by the end of 2021
    • Finalized plan for commercial grade material and initiated development of comparability protocol to support BLA/MAA filing

    TSHA-101 for GM2 gangliosidosis: the first bicistronic gene therapy in clinical development designed to deliver two genes – HEXA and HEXB, comprising the alpha and beta sub-units of β-Hexosaminidase A, intrathecally for the treatment of GM2 gangliosidosis, also called Tay-Sachs or Sandhoff disease

    • Based on natural history data, 2% to 4% Hex A enzyme activity in plasma normalizes survival and significantly improves clinical phenotype of GM2 gangliosidosis
    • Preclinical data demonstrated intrathecal delivery of TSHA-101 was safe and well-tolerated in GM2 knockout mice
    • Expect preliminary clinical safety data and HEX A enzyme activity in plasma and CSF for TSHA-101 in GM2 gangliosidosis in December 2021 from ongoing Canadian study, where Hex A enzyme activity level of at least 5% in plasma would be considered disease modifying based on natural history data
    • Due to severity of the disease and unmet medical need, currently assessing the need for US study to support regulatory filing

    AAV9 Gene Replacement for CLN7 disease: an investigational AAV9 intrathecally dosed gene replacement therapy designed to deliver a full-length copy of the CLN7 gene to potentially treat CLN7 disease, a rapidly progressing rare lysosomal storage disease with no approved treatments. The first-generation construct is currently in clinical development with the next-generation construct anticipated to have improved potency, safety, packaging efficiency and manufacturability.

    • Toxicology studies in wild type rodents demonstrated safety and tolerability of intrathecal administration of the first-generation construct across all dose levels and time points
    • Preliminary clinical safety data for the first patient in history to be intrathecally dosed at 1.0x1015 total vg with the first-generation construct expected by December 2021
    • Completion of next-generation construct by year-end 2021, with initiation of a planned pivotal trial in 2022 using next-generation construct with reference to human proof-of-concept clinical data generated from the first-generation construct
    • Commercial-grade GMP material for next-generation construct expected in 2022

    TSHA-118 in CLN1 disease: a self-complementary AAV9 viral vector designed to express a human codon-optimized CLN1 transgene to potentially treat CLN1 disease, a rapidly progressing rare lysosomal storage disease with no approved treatments

    • Preclinical data demonstrated that TSHA-118 was safe and well tolerated following intrathecal administration in CLN1 knockout mice
    • In preclinical models, TSHA-118-treated mice demonstrated supraphysiological levels of active PPT1 enzyme with no associated adverse effects, suggesting a wide therapeutic window for clinical dosing
    • Currently open IND
    • Additional CTA filing submitted
    • Initiation of a Phase 1/2 clinical trial by year-end 2021
    • Preliminary clinical safety and PPT1 enzyme activity data expected in first half of 2022

    TSHA-102 in Rett syndrome: a self-complementary AAV9 gene therapy in development for a severe neurodevelopmental disorder, designed to deliver miniMECP2, as well as a novel miRARE platform that regulates transgene expression genotypically on a cell-by-cell basis

    • In preclinical animal models, intrathecal myc-tagged TSHA-102 was not associated with early death and did not cause adverse behavioral side effects in wild type mice demonstrating appropriate downregulation of miniMECP2 protein expression as compared to unregulated MECP2 gene therapy constructs
    • Preclinical data demonstrated that miRARE regulated transgene expression genotypically on a cell-by-cell basis and improved the safety of TSHA-102 without compromising efficacy in juvenile mice
    • Recently obtained pharmacology data demonstrated improvement in survival, and respiratory and motor functions in disease relevant mouse models. Data to be shared at a later date
    • Preliminary data from a GLP toxicology study in non-human primates demonstrated no adverse findings at the highest dose tested suggesting that the miRARE platform is successfully downregulating MECP2 expression to within normal physiological levels
    • Submission of IND/CTA filing in November 2021
    • Initiation of Phase 1/2 clinical trial by year-end 2021
    • Preliminary clinical data expected by year-end 2022

    Third Quarter 2021 Financial Highlights

    Research and Development (R&D) Expenses: Research and development expenses were $39.5 million for the three months ended September 30, 2021, compared to $11.1 million for the three months ended September 30, 2020. The $28.4 million increase was primarily attributable to an increase of $14.5 million of expenses incurred in research and development manufacturing and other raw material purchases, which included cGMP batches produced by Catalent and UT Southwestern. There was an increase in employee compensation expenses of $10.7 million, which included $1.9 million of non-cash stock-based compensation, and $4.9 million in third-party research and development expenses, which includes clinical trial CRO activities, GLP toxicology studies, and consulting for regulatory and clinical studies. This was partially offset by a decrease in licensing fees of $1.7 million.

    General and Administrative (G&A) Expenses: General and administrative expenses were $11.2 million for the three months ended September 30, 2021, compared to $4.0 million for the three months ended September 30, 2020. The increase of approximately $7.2 million was primarily attributable to $4.3 million of incremental compensation expense, which included $1.8 million of non-cash stock-based compensation. There was an increase of $2.9 million mainly in professional fees related to legal, insurance, investor relations/communications, accounting, personnel recruiting, market research, and patient advocacy activities.

    Net loss: Net loss for the three months ended September 30, 2021 was $51.2 million or $1.35 per share, as compared to a net loss of $15.0 million, or $1.28 per share, for the three months ended September 30, 2020.

    Cash and cash equivalents: As of September 30, 2021, Taysha had $188.8 million in cash and cash equivalents.

    Conference Call and Webcast Information

    Taysha management will hold a conference call and webcast today at 8:00 am ET / 7:00 am CT to review its financial and operating results and to provide a corporate update. 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 13723855. 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 to build an extensive, AAV gene therapy pipeline focused on both rare and large-market indications. Together, we leverage our fully integrated platform—an engine for potential new cures—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 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 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, the potential market opportunity for these product candidates, and our corporate growth plans. 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, 2020 and our Quarterly Report on Form 10-Q for the quarter ended September 30, 2021, 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. Such risks may be amplified by the impacts of the COVID-19 pandemic. 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 Statements of Operations

    (in thousands, except share and per share data)

    (Unaudited)

     

    For the Three Months

    Ended September 30,

    For the Nine Months

    Ended September 30,

    2021

    2020

    2021

    2020

    Operating expenses:
    Research and development

    $

    39,528

     

    $

    11,057

     

    $

    94,025

     

    $

    19,633

     

    General and administrative

     

    11,153

     

     

    3,984

     

     

    29,518

     

     

    5,002

     

    Total operating expenses

     

    (50,681

    )

     

    15,041

     

     

    123,543

     

     

    24,635

     

    Loss from operations

     

    (50,681

    )

     

    (15,041

    )

     

    (123,543

    )

     

    (24,635

    )

    Other income (expense):
    Change in fair value of preferred stock tranche liability

     

    -

     

     

    -

     

     

    -

     

     

    (17,030

    )

    Interest income

     

    37

     

     

    -

     

     

    143

     

     

    -

     

    Interest expense

     

    (543

    )

     

    (1

    )

     

    (737

    )

     

    (28

    )

    Total other expense, net

     

    (506

    )

     

    (1

    )

     

    (594

    )

     

    (17,058

    )

    Net loss

    $

    (51,187

    )

    $

    (15,042

    )

    $

    (124,137

    )

    $

    (41,693

    )

     
    Net loss per common share, basic and diluted

    $

    (1.35

    )

    $

    (1.28

    )

    $

    (3.31

    )

    $

    (3.73

    )

    Weighted average common shares outstanding, basic and diluted

     

    38,003,954

     

     

    11,733,170

     

     

    37,495,537

     

     

    11,176,429

     

     

    Taysha Gene Therapies, Inc.

    Condensed Consolidated Balance Sheet Data

    (in thousands, except share and per share data)

    (Unaudited)

     

    September 30,

    2021

    December 31,

    2020

    ASSETS
    Current assets:
    Cash and cash equivalents

    $

    188,785

     

    $

    251,253

     

    Prepaid expenses and other current assets

     

    8,385

     

     

    6,626

     

    Total current assets

     

    197,170

     

     

    257,879

     

    Restricted cash

     

    2,628

     

     

    -

     

    Deferred lease asset

     

    691

     

     

    715

     

    Property, plant and equipment, net

     

    40,553

     

     

    287

     

    Total assets

    $

    241,042

     

    $

    258,881

     

     
    LIABILITIES AND STOCKHOLDERS' EQUITY
    Current liabilities
    Accounts payable

    $

    22,051

     

    $

    1,994

     

    Accrued expenses and other current liabilities

     

    21,163

     

     

    5,135

     

    Total current liabilities

     

    43,214

     

     

    7,129

     

    Build-to-suit lease liability

     

    26,607

     

     

    -

     

    Term Loan, net

     

    27,812

     

     

    -

     

    Other non-current liabilities

     

    3,015

     

     

    450

     

    Total liabilities

     

    100,648

     

     

    7,579

     

     
    Stockholders' equity
    Preferred stock, $0.00001 par value per share; 10,000,000 shares authorized and no shares issued and outstanding as of September 30, 2021 and December 31, 2020

     

    -

     

     

    -

     

    Common stock, $0.00001 par value per share; 200,000,000 shares authorized and 38,473,945 and 37,761,435 issued and outstanding as of September 30, 2021 and December 31, 2020

     

    -

     

     

    -

     

    Additional paid-in capital

     

    325,657

     

     

    312,428

     

    Accumulated deficit

     

    (185,263

    )

     

    (61,126

    )

    Total stockholders' equity

     

    140,394

     

     

    251,302

     

    Total liabilities and stockholders' equity

    $

    241,042

     

    $

    258,881

     

     

    View Full Article Hide Full Article
  2. Stifel 2021 Virtual Healthcare Conference on November 15, 2021 at 11:20 am ET

    Jefferies London Healthcare Conference November 17, 2021 at 12:20 pm GMT

    4th Annual Evercore ISI HealthCONx Virtual Conference on November 30, 2021 at 4:20 pm ET

    Taysha Gene Therapies, Inc. (NASDAQ:TSHA), a patient-centric, pivotal-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) in both rare and large patient populations, today announced its participation in the Stifel 2021 Virtual Healthcare Conference, Jefferies London Healthcare Conference, and the 4th Annual Evercore ISI HealthCONx Virtual Conference.

    Conference Details:

    Event:

    Stifel 2021 Virtual Healthcare Conference on November 15, 2021 at 11:20 am ET

    Jefferies London Healthcare Conference November 17, 2021 at 12:20 pm GMT

    4th Annual Evercore ISI HealthCONx Virtual Conference on November 30, 2021 at 4:20 pm ET

    Taysha Gene Therapies, Inc. (NASDAQ:TSHA), a patient-centric, pivotal-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) in both rare and large patient populations, today announced its participation in the Stifel 2021 Virtual Healthcare Conference, Jefferies London Healthcare Conference, and the 4th Annual Evercore ISI HealthCONx Virtual Conference.

    Conference Details:

    Event:

     

    Stifel 2021 Virtual Healthcare Conference

    Date:

     

    November 15, 2021

    Time:

     

    11:20 am ET

    Format:

     

    Fireside Chat

    Participants:

     

    RA Session II, President, Founder and Chief Executive Officer

     

     

    Dr. Suyash Prasad, Chief Medical Officer and Head of R&D

     

     

    Kamran Alam, Chief Financial Officer

     

     

    Dr. Kimberly Lee, SVP, Corporate Communications and Investor Relations

     

     

     

    Event:

     

    Jefferies London Healthcare Conference

    Date:

     

    November 17, 2021

    Time:

     

    12:20 pm GMT

    Format:

     

    Fireside Chat

    Participant:

     

    RA Session II, President, Founder and Chief Executive Officer

     

     

    Dr. Suyash Prasad, Chief Medical Officer and Head of R&D

     

     

    Kamran Alam, Chief Financial Officer

     

     

    Dr. Kimberly Lee, SVP, Corporate Communications and Investor Relations

     

     

     

    Event:

     

    4th Annual Evercore ISI HealthCONx Virtual Conference

    Date:

     

    November 30, 2021

    Time:

     

    4:20 pm ET

    Format:

     

    Fireside Chat

    Participant:

     

    RA Session II, President, Founder and Chief Executive Officer

     

     

    Dr. Suyash Prasad, Chief Medical Officer and Head of R&D

     

     

    Kamran Alam, Chief Financial Officer

     

     

    Dr. Kimberly Lee, SVP, Corporate Communications and Investor Relations

    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 to build an extensive, AAV gene therapy pipeline focused on both rare and large-market indications. Together, we leverage our fully integrated platform—an engine for potential new cures—with a goal of dramatically improving patients' lives. More information is available at www.tayshagtx.com.

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  3. Taysha Gene Therapies, Inc. (NASDAQ:TSHA), a patient-centric, pivotal-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) in both rare and large patient populations, today announced that it will report its financial results for the third quarter ended September 30, 2021, and host a corporate update conference call and webcast on Wednesday, November 10, 2021, at 8:00 AM Eastern Time.

    Conference Call Details
    Wednesday, November 10, at 8:00 AM Eastern Time / 7:00 AM Central Time

    Toll Free:

    877-407-0792

    International:

    201-689-8263

    Conference ID:

    13723855

    Webcast:

    https://ir.tayshagtx.com/news-events/events-presentations

    Taysha Gene Therapies, Inc. (NASDAQ:TSHA), a patient-centric, pivotal-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) in both rare and large patient populations, today announced that it will report its financial results for the third quarter ended September 30, 2021, and host a corporate update conference call and webcast on Wednesday, November 10, 2021, at 8:00 AM Eastern Time.

    Conference Call Details
    Wednesday, November 10, at 8:00 AM Eastern Time / 7:00 AM Central Time

    Toll Free:

    877-407-0792

    International:

    201-689-8263

    Conference ID:

    13723855

    Webcast:

    https://ir.tayshagtx.com/news-events/events-presentations

    An archived version of the webcast will be available on Taysha's 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 to build an extensive, AAV gene therapy pipeline focused on both rare and large-market indications. Together, we leverage our fully integrated platform—an engine for potential new cures—with a goal of dramatically improving patients' lives. More information is available at www.tayshagtx.com.

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  4. AAV-mediated UBE3A gene replacement recapitulates endogenous isoform ratios by replacing both the short and long isoforms of UBE3A in key regions of the brain, leading to improvements in motor learning, behavior outcomes, and seizure phenotypes in Angelman mouse models

    Novel construct, originally developed in the laboratories of Dr. Ben Philpot and Dr. Steven Gray, packages both short and long isoforms of UBE3A into a single viral vector, which is expected to confer significant advantages over approaches that express only one of the isoforms

    Proof-of-concept preclinical data support further study of UBE3A gene replacement therapy as a potentially safe and effective treatment for Angelman syndrome

    Adding to an existing vectorized shRNA knockdown

    AAV-mediated UBE3A gene replacement recapitulates endogenous isoform ratios by replacing both the short and long isoforms of UBE3A in key regions of the brain, leading to improvements in motor learning, behavior outcomes, and seizure phenotypes in Angelman mouse models

    Novel construct, originally developed in the laboratories of Dr. Ben Philpot and Dr. Steven Gray, packages both short and long isoforms of UBE3A into a single viral vector, which is expected to confer significant advantages over approaches that express only one of the isoforms

    Proof-of-concept preclinical data support further study of UBE3A gene replacement therapy as a potentially safe and effective treatment for Angelman syndrome

    Adding to an existing vectorized shRNA knockdown approach to unsilence the paternal UBE3A allele, UBE3A gene replacement bolsters Taysha's pipeline in Angelman syndrome and strengthens its position as a world-leader in developing gene therapies for monogenic CNS disease

    Angelman syndrome investor day on Tuesday, October 26, 2021, 10:00 am-1:00 pm ET

    Taysha Gene Therapies, Inc. (NASDAQ:TSHA), a patient-centric, pivotal-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) in both rare and large patient populations, today announced the publication of new preclinical data for an AAV-mediated UBE3A gene replacement approach for the treatment of Angelman syndrome in the Journal of Clinical Investigation Insight (JCI Insight).

    Angelman syndrome (AS) is a monogenic neurodevelopmental disorder caused by deletions or mutations in the maternal ubiquitin protein ligase E3A (UBE3A) gene. UBE3A encodes both short and long protein isoforms. Whereas the short isoform is most abundant, clinical data indicate that long UBE3A isoforms also contribute to healthy brain development and function. Common signs and symptoms of Angelman syndrome typically appear early in childhood and include intellectual and developmental disabilities, walking and balance disorders, gastrointestinal issues, seizures, and little to no speech. There are currently no approved treatments for Angelman syndrome and current interventions are focused on managing medical and developmental issues. Angelman syndrome impacts approximately 55,000 patients in the United States and in Europe.

    Ben Philpot, Ph.D., Associate Director of the UNC Neuroscience Center, said, "The highly evolutionarily conserved expression of both the short and long isoforms of UBE3A suggests their importance, and we have identified an approach capable of packaging both isoforms into a single viral vector. To recapitulate natural human UBE3A isoform levels, we ensured expression of short and long isoforms at the endogenous 3 to 1 ratio, as well as the selective expression in neurons where UBE3A is lost, thus limiting peripheral toxicity. We are highly encouraged by our preclinical data demonstrating recovery in motor performance, epilepsy, and anatomical markers, supporting the potential of UBE3A gene replacement for the treatment of Angelman syndrome. We look forward to continued advancement of this approach."

    "The UBE3A gene replacement approach is unique from our first Angelman syndrome candidate, which uses vectorized RNA-mediated knockdown to unsilence the paternal copy of the UBE3A gene by targeting the antisense transcript responsible for silencing the gene," said Suyash Prasad, MBBS, M.Sc., MRCP, MRCPCH, FFPM, Chief Medical Officer and Head of Research and Development of Taysha. "We are encouraged by the improvements in motor learning, behavior outcomes, and seizure phenotypes achieved with UBE3A gene replacement in mouse models of Angelman syndrome. We believe both the gene replacement and RNA-mediated knockdown strategies position Taysha as a world-leader in the discovery of treatments for Angelman syndrome, a significant neurodevelopmental disorder with no currently approved treatments."

    Taysha's UBE3A gene replacement therapy is a cerebrospinal fluid-delivered AAV vector enabling dual isoform UBE3A expression for the treatment of Angelman syndrome. It was originally developed in the laboratories of Dr. Ben Philpot and Taysha's Chief Scientific Advisor, Dr. Steven Gray. In preclinical mouse models of Angelman syndrome, AAV-mediated UBE3A gene replacement recapitulated endogenous UBE3A isoform expression and UBE3A subcellular expression in neurons. Anatomical and behavioral phenotypes, including nest building, motor performance and seizure phenotypes, were recovered following treatment, providing proof-of-concept preclinical data supporting further study of UBE3A gene replacement therapy as a potentially safe and effective treatment for Angelman syndrome.

    Dr. Philpot will present these and additional preclinical data for Taysha's AAV-mediated UBE3A gene replacement approach at the Company's upcoming Angelman investor day on Tuesday, October 26th. Please register for the event here. The virtual event will feature presentations from Key Opinion Leaders who will provide an overview of Angelman syndrome, discuss natural history data, both Taysha approaches for gene therapy treatment mechanisms, and Taysha's clinical development strategy.

    *Disclosures - UT Southwestern and Dr. Gray hold financial interest in Taysha. Dr. Gray serves as Chief Scientific Advisor for Taysha. UT Southwestern maintains a financial interest in Taysha unrelated to this research.

    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 to build an extensive, AAV gene therapy pipeline focused on both rare and large-market indications. Together, we leverage our fully integrated platform—an engine for potential new cures—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," 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 TSHA-106, 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, TSHA-106's eligibility for accelerated approval in the United States and Europe, 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 these 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 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, 2020, and our Quarterly Report on Form 10-Q for the quarter ended June 30, 2021, 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. Such risks may be amplified by the impacts of the COVID-19 pandemic. 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.

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  5. Jefferies Gene Therapy & Editing Summit on October 28 at 11:00 am ET

    Taysha Gene Therapies, Inc. (NASDAQ:TSHA), a patient-centric, pivotal-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) in both rare and large patient populations, today announced its participation in a fireside chat at the Jefferies Gene Therapy & Editing Summit.

    Conference Details:

    Event:

    Jefferies Gene Therapy & Editing Summit

    Date:

    Thursday, October 28, 2021

    Time:

    11:00 am ET

    Format:

    Fireside Chat

    Participants:

    RA Session II, President, Founder and Chief Executive Officer

     

    Dr. Suyash Prasad, Chief…

    Jefferies Gene Therapy & Editing Summit on October 28 at 11:00 am ET

    Taysha Gene Therapies, Inc. (NASDAQ:TSHA), a patient-centric, pivotal-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) in both rare and large patient populations, today announced its participation in a fireside chat at the Jefferies Gene Therapy & Editing Summit.

    Conference Details:

    Event:

    Jefferies Gene Therapy & Editing Summit

    Date:

    Thursday, October 28, 2021

    Time:

    11:00 am ET

    Format:

    Fireside Chat

    Participants:

    RA Session II, President, Founder and Chief Executive Officer

     

    Dr. Suyash Prasad, Chief Medical Officer and Head of R&D

     

    Kamran Alam, Chief Financial Officer

     

    Dr. Kimberly Lee, SVP, Corporate Communications and Investor Relations

    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 to build an extensive, AAV gene therapy pipeline focused on both rare and large-market indications. Together, we leverage our fully integrated platform—an engine for potential new cures—with a goal of dramatically improving patients' lives. More information is available at www.tayshagtx.com.

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