1. What is the Chemxpert database?

Chemxpert database is a techno-commercial information platform developed and operated by Lifescience Intellipedia Pvt. Ltd. Our data provides valuable insights regarding sourcing, marketing, development, and regulatory functions for the entire Lifescience industry. The different industries covered by our data encompass APIs, FDFs, pharma intermediates, paints, dyes, polymers, biotechnology, biochemical, food additives, nutraceuticals, etc.

2. How can I subscribe to the Chemxpert database?

You can gain access to the Chemxpert database after subscribing to it by visiting this link: - https://chemxpert.com/pricing

3. How many countries' data is available on your portal?

The Chemxpert database covers more than 200 countries. We are working in the direction of expanding our data coverage in more countries.

4. What are the different sources you use for obtaining information?

The different sources we use for data collection are as follows: - • Primary researc • DMF, ANDA, & CEP list • Conferences, Exhibitions, & Tradeshow • Company annual reports • DMF, ANDA, & CEP list • Subject matter experts & KOL survey. Apart from these, our other valuable sources include application-wise manufacturers list, subscribed and non-subscribed databases, corporate partners, US FDA Orange book, publicly available sources, industry associates, national statistics, regulatory websites, etc. You can gain access to the Chemxpert database after subscribing to it by visiting this link: -https://chemxpert.com/sources

5. What is the expected time for account activation?

Your Chemxpert account will be activated instantly if the payment is made through our website. If you choose to use RTGS, NEFT, or IMPS systems for payment, your account will be activated within 24 hours.

06 Oct 2025
Admin
News Article

Taysha Gene Therapies’ TSHA-102 Receives FDA Breakthrough Therapy Designation for Rett Syndrome

Taysha Gene Therapies announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to TSHA-102, its investigational gene therapy for Rett syndrome.

Multiple Regulatory Designations

TSHA-102 has secured several regulatory recognitions, including:

FDA: Breakthrough Therapy, Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug, and Rare Pediatric Disease designations
European Commission: Orphan Drug designation
UK regulator: Innovative Licensing and Access Pathway (ILAP) designation

These designations highlight the therapy’s potential to address a serious unmet need in Rett syndrome, a rare genetic neurodevelopmental disorder caused by mutations in the MECP2 gene.

About Rett Syndrome

Rett syndrome primarily affects females and is characterized by:

Loss of communication skills and impaired hand function
Developmental regression and motor difficulties
Respiratory issues, seizures, and intellectual disability

There are no approved disease-modifying therapies, and it is estimated to affect 15,000–20,000 patients across the US, EU, and UK.

TSHA-102 and Clinical Progress

TSHA-102 is an intrathecally delivered AAV9 gene therapy designed as a one-time treatment to deliver a functional form of MECP2 to cells in the central nervous system.

Clinical data from Part A of the ongoing REVEAL Phase 1/2 trials showed a favorable safety profile and a 100% response rate at the May 2025 cutoff, with patients achieving at least one developmental milestone.
Dose-related improvements were observed in motor behavior and clinician-assessed global improvement.

Pivotal REVEAL Trial

The REVEAL pivotal trial will evaluate a single high dose of TSHA-102 in 15 females aged 6 to under 22 years with Rett syndrome in the developmental plateau stage.

Primary endpoint: Proportion of patients achieving one or more developmental milestones in communication, fine motor, or gross motor domains
Design: Each patient serves as their own control, with standardized, video-reviewed assessments by independent blinded raters
Interim analysis: Conducted at six months, potentially supporting a future Biologics License Application (BLA)

Previous findings indicated that 83% of patients receiving high-dose treatment achieved milestone gains within six months.

Let’s apply Data-Driven Pricing to Your APIs

Sick and tired of always wondering if you are being asked to pay the right price for your APIs? This empowers you with the answers you need to make the right decisions in the Global API market.

Chemxpert Database is one of the biggest and most comprehensive directories of pharma and chemicals, manufacturers, suppliers and information. Provided with current information on prices, demand and transactions, it gives you instant feedback on whether you are buying what is right and at the right time.

Start using market intelligence today and allow yourself to be in control in the API market.

Check it out today and make more informed sourcing decisions! Learn More!