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gene-therapy: Archive

Zacks Equity Research

CRISPR Therapeutics Q4 Earnings and Revenues Surpass Estimates

CRSP reports encouraging fourth-quarter results. The commercial launch of the first CRISPR-based gene therapy, Casgevy, is progressing well.

ALNYPositive Net Change BMRNNegative Net Change VRTXNegative Net Change CRSPNegative Net Change

Zacks Equity Research

RARE Reports New Upbeat Data From Sanfilippo Syndrome Drug Study

Ultragenyx announces new safety and efficacy data from its pivotal study of its investigational candidate, UX111, for patients with Sanfilippo syndrome type A.

ALNYPositive Net Change BMRNNegative Net Change RARENegative Net Change CSTLNegative Net Change

Sundeep Ganoria

SRPT Stock Rises 23% in a Year: Time to Buy, Hold or Sell?

The upside in Sarepta's share price can be attributed to the encouraging sales performance of its DMD gene therapy, which has demonstrated blockbuster potential.

RHHBYPositive Net Change SRPTPositive Net Change ARWRNegative Net Change

Zacks Equity Research

RARE Submits BLA to the FDA for Sanfilippo Syndrome Gene Therapy

Ultragenyx submits a BLA to the FDA seeking the accelerated approval of UX111 for patients with Sanfilippo syndrome type A in the United States.

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Ekta Bagri

4 Biotech Stocks Most Wall Street Analysts Are Bullish About

Here, we present four biotech stocks, namely SRPT, ADMA, EXEL and ALNY, which most Wall Street analysts are optimistic about. These companies have upside potential for 2025, backed by strong fundamentals.

ALNYPositive Net Change EXELNegative Net Change SRPTPositive Net Change ADMANegative Net Change

Zacks Equity Research

SRPT Finishes Enrolment in Late-Stage Study for Rare Muscular Disorder

The phase III EMERGENE study evaluates Sarepta's experimental gene therapy for an ultra-rare form of muscle disorder for which there is no treatment beyond symptom management.

RHHBYPositive Net Change SRPTPositive Net Change CTMXNegative Net Change CSTLNegative Net Change

Ekta Bagri

3 Biotech Stocks With Promising Gene Therapies to Watch Out in 2025

We have picked three biotech stocks with promising gene therapies in their portfolios/pipelines that are likely to perform well in 2025.

SRPTPositive Net Change VYGRNegative Net Change CRSPNegative Net Change