Neukene在Baird治疗Rett综合征的基因治疗方面取得了新的超越者

2024-06-11 23:02

Baird started Neurogene (NASDAQ:NGNE) off at outperform as its Rett syndrome candidate, NGN-401, has shown promise with early data and the asset was recently selected by the U.S. FDA for a pilot program to accelerate development of rare disease therapies.
The firm has a $54 price target (~45% upside based on June 11 close).
Analyst Joel Beatty noted that NGN-401 uses the company's EXACT technology, which provides controlled transgene expression. Neurogene's website states that too much expression can cause toxicity, while too little can cause disease.
He added that preclinical data showed that NGN-401 was "able to cause the production of enough MeCP2 protein to greatly improve survival and function."
Beatty also sees NGN-401 as more promising than Taysha Gene Therapies' (TSHA) TSHA-102, which, like NGN-401, is in phase 1/2. This is because mice born with the minigene used in TSHA-102 had some motor abnormalities and weighed significantly less than the full-length gene used in NGN-401. Also, intracerebral ventricular delivery used with NGN-401 "appears to lead [to] far higher distribution and expression in the brain than the intrathecal (IT) delivery used with TSHA-102."
He projects NGN-401 could have a one-time price of $2.3M. Given an estimated U.S. prevalence of 6,000-9,000 individuals, this could account for a market opportunity as large as $14B-$21B.

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