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| At week-35, she also showed a sustained improvement in her ability to grasp with her dominant hand |
| She also sustained improvement in autonomic function at week-19 with improvements in breathing dysrhythmias, including hyperventilation and reduced apneic spells |
| Overall we are highly encouraged by the safety profile and the durable response reported in these long-term data in both adult patients treated with the low dose of TSHA-102 |
| With a significant focus on execution in the year ahead, we believe our team is well positioned to continue to accelerate the development of TSHA-102 |
| Sean Nolan If you're referring to like CGI-I would say this -- is that what you're talking about? Yanan Zhu Sorry, sorry, CGI-I, I fully appreciate even maintenance of the prior numbers according to our check with the doctors, that's a very, very encouraging sign to have those minimum improved ratings maintained following in a follow-up and also to have the much improved for the other patient maintained |
| These continued improvements in both adult patients with advanced Stage 4 Rett syndrome, coupled with the IDMC's approval to dose the second pediatric patient following a review of the initial clinical data from the first pediatric patient dose with TSHA-102 reinforced the transformative potential of TSHA-102 across a broad population of patients with Rett syndrome |
| Lastly, we are pleased to share that we have strengthened our clinical and regulatory leadership with the promotion of Dr |
| Therefore, it was very exciting when we announced the encouraging initial impact that TSHA-102 appeared to have across multiple clinical domains in the first two adult patients treated as early as four weeks following the treatment that we reported in November 2023 |
| We believe the safety profile and continued improvements observed even at reduced steroid levels in both adult patients with advanced Stage 4 Rett syndrome support the durability and transformative potential of TSHA-102 across multiple genotypes of Rett syndrome |
| We believe the safety profile and these continued improvements across multiple clinical domains, even at reduced steroid levels, in both adult patients with advanced stage four Rett syndrome, support the durability and transformative potential of TSHA-102 across multiple genotypes of Rett syndrome and further validate our construct |
| Overall, we are highly encouraged by the safety profile and long-term efficacy reported in the first two adult patients, as well as IDMC's approval to dose the second pediatric patient following review of the initial six-week clinical data from the first pediatric patient dosed with TSHA-102 |
| These sustained improvements in hand function, 35-weeks following treatment, which are not observed in the natural history of Rett syndrome, are very encouraging and support the potential of TSHA-102 to bring meaningful therapeutic benefit to patients and caregivers |
| The sustained improvement in hand stereotypies at week-19 post-treatment is encouraging and it provides new opportunities for fine motor skill learning |
| In addition to the positive safety data, we are encouraged by the longer term safety profile observed |
| These new improvements observed at week-35 post-treatment are highly encouraging as alternative and augmentative communication speech output technologies activated by Eyegaze can be leveraged by patients and families with Rett syndrome as a supplement to or replacement for natural speech |
| So we're quite encouraged by what we've seen in that aspect of things |
| We are pleased that TSHA-102 recently received ILAP designation from the U.K |
| We are not surprised, but nonetheless pleased to highlight today that patient improvements were maintained or further improved in the absence of steroids |
| I would say we're -- we continue to be very encouraged of the pathway that we're on to be further informed in the coming quarters that can help us with the FDA |
| Importantly, these continued improvements were reported at week 35 following completion of the steroid taper for the first patient and at week 19 at decreased steroid levels for the second patient |
| We are highly encouraged by the safety profile and durable response reported at reduced steroid levels and the longer-term data from both patients in the low dose of our Reveal adolescent and adult trial |
| The patient also sustained improvements in socialization and communication through week-19 with an increased interest in social communication and activities, including increased response to spoken words and eye contact |
| As you can see, we have made exciting progress across our TSHA-102 program over the past year |
| Building on the momentum from 2023, we believe 2024 is poised to be a transformational year for the company |
| Importantly, we believe these accomplishments enable us to focus our efforts this year on generating critical, long-term clinical data in a broad range of ages and stages of Rett syndrome patients across multiple geographies |
| By increasing MeCP2 levels in MeCP2 deficient cells and maintaining healthy levels of MeCP2 output in normal cells, TSHA-102 has demonstrated the ability to produce and maintain safe transgene expression in the CNS in preclinical models |
| Therefore, it's encouraging that the patient also showed a sustained improvement in circulation at week-35 post-treatment with the patient's hands and feet restored to normal temperature and color, whereas before treatment the hands and feet were usually cold and blue based on the principal investigator's clinical observations |
| The sustained and new improvements in both adult patients with Advanced Aged Force Syndrome coupled with the initial six-week clinical data from the first pediatric patient that was reviewed by the IDMC, support the therapeutic potential of TSHA-102 for a broad population of patients with Rett syndrome |
| Those are great achievements |
| Importantly, we continue to see a durable response and we are seeing sustained improvement in the absence or reduction of steroid levels |
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| Her ability to reach and grasp objects was weak |
| Put another way, it's possible that the steroids somewhat masked benefits until they're either reduced significantly or further withdrawn |
| Difficulty in communication, including loss of speech, is one of the most prominent symptoms of Rett syndrome and is a key area of concern for caregivers as it directly interferes with the patient's ability to communicate their needs and express their interest |
| Additionally, the patient had limited body movement requiring constant back support and had lost fine and gross motor function early in childhood |
| Additionally, poor perfusion of the extremities is a characteristic sign in patients with Rett syndrome that is thought to result from dysautonomia, meaning it's controlled by the autonomic nervous system |
| Progressive loss of hand function is a hallmark characteristic of Rett syndrome and a key concern for caregivers that impacts a patient's ability to communicate and impede daily activities, but ultimately limits independence |
| She has had a bit of an issue with tolerating the steroids |
| Recall, when we initiated the REVEAL trial, there were low expectations of efficacy for the Stage 4 adult population among KOLs in the Rett syndrome community, due to the advanced and relentless progression of disease |
| So this patient had, I think, eight to 16 seizures a month, and other than one seizure day 13 post treatment, the patient has had no new seizures and also the use of a combination antiepileptic meds have dropped by 25% |
| This disorder is characterized by loss of communication and hand function, slowing and regression of development, motor and respiratory impairment, seizures, intellectual disabilities, and shortened life expectancy |
| Various risks may Taysha's actual results to differ materially from those stated or implied in such forward-looking statements |
| So -- but this patient and Patient 1 had serious sleep abnormalities |
| Net loss for the full-year ended December 31, 2023 was $111.6 million or $0.96 per share, as compared to a net loss of $166 million or $3.78 per share for the full-year ended December 31, 2022 |
| There is a significant unmet medical need with Rett syndrome caused by a pathogenic, likely pathogenic MECP2 mutation, afflicting between 15,000 to 20,000 patients in the U.S., EU, and U.K., and a high burden of care associated |
| She had lost the ability to sit or stand by eight years of age as documented in the patient's medical history |
| Additionally, the patient experienced frequent hyperventilation episodes and had a history of frequent seizures |
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