{"id":49791,"date":"2023-08-17T18:50:43","date_gmt":"2023-08-17T22:50:43","guid":{"rendered":"https:\/\/ifintechworld.com\/news\/taysha-gene-therapies-rett-syndrome-data-makes-this-a-must-watch-nasdaqtsha\/"},"modified":"2023-08-17T18:50:47","modified_gmt":"2023-08-17T22:50:47","slug":"taysha-gene-therapies-rett-syndrome-data-makes-this-a-must-watch-nasdaqtsha","status":"publish","type":"post","link":"https:\/\/ifintechworld.com\/?p=49791","title":{"rendered":"Taysha Gene Therapies: Rett Syndrome Data Makes This A Must Watch (NASDAQ:TSHA)"},"content":{"rendered":"
\n

<\/picture>
<\/figcaption><\/figure>\n<\/p>\n

Taysha Gene Therapies<\/strong> (NASDAQ:TSHA<\/span>) has made great progress recently in advancing its gene therapy known as TSHA-102 for the treatment of patients with Rett Syndrome. Despite only having data from one patient so far, with a second expected to start dosing in Q3 of 2023, the data shown thus far is impressive. The biotech noted that it intends to provide quarterly updates for its clinical programs, thus data from the second patient recruited into this phase 1\/2 REVEAL trial, could be released by Q4 of 2023. If the second patient achieves a similar or better outcome than the first patient, then it will be confirmation that TSHA-102 may be highly suitable to treat patients with Rett Syndrome.<\/span><\/p>\n

Besides such a catalyst in the coming months, this biotech has another gene therapy candidate being advanced, which is TSHA-120. This other gene therapy candidate is being advanced for the treatment of patients with Giant Axonal Neuropathy [GAN]. Taysha is <\/span>gearing up to meet with the FDA in Q3 of 2023<\/strong>, whereby it and the agency will discuss a potential regulatory pathway forward for the use of TSHA-120 for the treatment of patients with GAN. Should this meeting conclude in a successful manner, then the company may be given the green light to initiate a pivotal study for this program. This remains to be seen, but this is another catalyst for traders\/investors to look forward to. Lastly, it was able to obtain a $150 million private placement financing to boost its cash on hand. It believes that with this recent financing transaction, that it would have enough cash on hand to fund its operations into Q3 of 2025.<\/span><\/p>\n

TSHA-102 Achieves Huge Improvement In First Patient With Rett Syndrome<\/h2>\n

Taysha Gene Therapies is advancing the use of gene therapy candidate TSHA-102 for the treatment of patients with Rett Syndrome. Rett Syndrome<\/strong> is a rare genetic neurological disorder by which severe impairment occurs in every aspect of an affected person’s life. It affects many functions of life such as: Speech, coordination, and motor movement. It occurs as a result of a gene mutation, which is first recognized in infancy. While this disorder primarily affects girls, it is also being identified in many males as well. Other problems associated with this disease are muscle movements and communication skills. The thing is that most babies with Rett Syndrome develop as expected in the first 6 months of their life, however, they then lose such skills previously learned. They lose the ability to be able to communicate properly, crawl, walk or use their hands as needed. It is expected that the Rett Syndrome market will hit $515.06 million by 2029<\/strong>. This is a pretty good market opportunity for any biotech that can capture this market. Thus far, the whole market lies at the foot of Acadia Pharmaceuticals<\/strong> (ACAD), which received FDA approval of its drug Daybue<\/strong> for the treatment of Rett Syndrome in adult and pediatric patients 2 years of age and older. One thing to note is that Daybue is the first and only drug approved for the treatment of this patient population. However, another biotech with potential to capture this Rett Syndrome indication might be Anavex Life Sciences<\/strong> (AVXL). That’s because it is gearing up to report top-line data from its potentially pivotal Anavex2-73-RS-003 phase 2\/3 EXCELLENCE pediatric clinical trial<\/strong>. Top-line results from this study are expected to be released in the 2nd half of 2023. If this study succeeds, then this might be another possible competitor that Taysha Gene Therapies may have to ultimately contend with.<\/p>\n

The thing is that the positive results that Taysha Gene Therapies was able to release, came from the phase 1\/2 REVEAL trial<\/strong>, which used gene therapy TSHA-102 for the treatment of 1 patient with Rett Syndrome. Despite this only being one patient, it was noted that they had achieved improvement in key efficacy measures. Such clinical efficacy measures where improvement was achieved is as follows:<\/p>\n