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gene-therapy: Archive

Zacks Equity Research

REGENXBIO's (RGNX) Hunter Syndrome Study Meets Goal, Stock Up

REGENXBIO (RGNX) gains 12% on positive top-line results from the phase I/II/III study of its investigational one-time gene therapy candidate, RGX-121, to treat pediatric patients with Hunter syndrome.

IMGNPositive Net Change PBYINegative Net Change RGNXNegative Net Change IMCRNegative Net Change

Zacks Equity Research

Vertex's (VRTX) Non-Opioid Pain Drug Meets Primary Goal

Three late-stage studies data show that Vertex's (VRTX) experimental non-opioid drug reduced pain across a 48-hour period. Based on this data, VRTX intends to submit an FDA filing by mid-2024.

VRTXPositive Net Change SRPTNegative Net Change AVXLPositive Net Change CRSPNegative Net Change

Zacks Equity Research

Sana Bio (SANA) Stock Rallies 105% in 3 Months: Here's Why

Sana Bio (SANA) rallies 105% after the company reports encouraging pipeline progress with its candidates, SC291 and SC262, for oncology and autoimmune indications.

PBYINegative Net Change ADMANegative Net Change ACADNegative Net Change SANANegative Net Change

Zacks Equity Research

Vertex (VRTX), CRSP's Casgevy Gets FDA Nod for Thalassemia

This is the second approval granted to Vertex (VRTX)/CRISPR's (CRSP) gene therapy Casgevy and comes just a month after the FDA approved the therapy for sickle cell disease.

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Zacks Equity Research

Solid Biosciences' (SLDB) DMD Drug Gets Orphan Tag, Stock Up

Solid Biosciences (SLDB) stock rises after the FDA grants the orphan drug designation to the company's novel gene-therapy candidate, SGT-003, for the treatment of Duchenne muscular dystrophy.

RHHBYNegative Net Change SRPTNegative Net Change PBYINegative Net Change SLDBNegative Net Change

Zacks Equity Research

Why Sarepta's (SRPT) Stock Price Moved Up 17% on Tuesday

Sarepta (SRPT) surges after its CEO made positive comments on the label expansion filing for the company's DMD gene therapy. The company expects a potential approval in August.

RHHBYNegative Net Change SRPTNegative Net Change CTMXPositive Net Change SLDBNegative Net Change