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gene-therapy: Archive

Zacks Equity Research

BioMarin (BMRN) Posts Q4 Earnings Beat, Upbeat Voxzogo Sales

BioMarin (BMRN) reports encouraging fourth-quarter results, with earnings and sales beating estimates. The rapid uptake of Voxzogo boosts revenues.

SNYPositive Net Change BMRNPositive Net Change ACETNegative Net Change PBYINegative Net Change

Zacks Equity Research

CRISPR (CRSP) Q4 Earnings Top on Collaboration Revenues, Stock Up

CRISPR (CRSP) gains on encouraging fourth-quarter 2023 results, beating both earnings and revenue estimates on the back of collaboration revenues received from Vertex.

VRTXNegative Net Change PBYINegative Net Change ADMAPositive Net Change CRSPPositive Net Change

Zacks Equity Research

Sarepta (SRPT) to Report Q4 Earnings: Will It Beat Estimates?

Investors' focus will likely be on Sarepta's (SRPT) product sales of its recently-approved DMD gene therapy when it reports fourth-quarter earnings.

RHHBYPositive Net Change SRPTPositive Net Change ARQTPositive Net Change CGEMPositive Net Change

Zacks Equity Research

Sarepta's (SRPT) DMD Drug sBLA Gets FDA's Priority Tag, Stock Up

Sarepta (SRPT) announces the FDA's acceptance of its efficacy supplement to the Elevidys BLA, seeking to expand the treatment label for its DMD drug. The stock gains 8%.

RHHBYPositive Net Change SRPTPositive Net Change ADMAPositive Net Change IMCRNegative Net Change

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.

PBYINegative Net Change RGNXPositive 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.

VRTXNegative Net Change SRPTPositive Net Change AVXLPositive Net Change CRSPPositive 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 ADMAPositive Net Change ACADPositive Net Change SANAPositive 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.

VRTXNegative Net Change SRPTPositive Net Change BLUEPositive Net Change CRSPPositive Net Change

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.

RHHBYPositive Net Change SRPTPositive Net Change PBYINegative Net Change SLDBPositive Net Change