Tag: gene therapy

Growing Awareness amongst Individuals on Benefits of Stem Cells to Fuel Global Induced Pluripotent Stem Cells Market

Brooklyn, NY, Sept. 13, 2016 (GLOBE NEWSWIRE) -- The first section of the report presents an overview of induced pluripotent stem cells. This section encapsulates the specifications, definition, and classification of induced pluripotent stem cells. Pluripotent stem cells refer to embryonic stem cells that have the ability to make each and every kind of adult…

AGTC Announces Financial Results and Business Update for the Quarter and Fiscal Year Ended June 30, 2016

GAINESVILLE, Fla., and CAMBRIDGE, Mass., Sept. 12, 2016 (GLOBE NEWSWIRE) -- Applied Genetic Technologies Corporation (NASDAQ:AGTC), a biotechnology company conducting human clinical trials of adeno-associated virus (AAV)-based gene therapies for the treatment of rare diseases, today announced financial results for the quarter ended June 30, 2016. “Over the past year we’ve continued to make progress…

Fibrocell Announces Closing of $18 Million Private Placement

EXTON, Pa., Sept. 08, 2016 (GLOBE NEWSWIRE) -- Fibrocell Science, Inc. (NASDAQ:FCSC) today reported the receipt of $18.1 million of gross proceeds from the sale of convertible promissory notes and warrants in its previously announced private placement financing. Fibrocell intends to use the proceeds of the financing for the continued pre-clinical and clinical development of…

AveXis Announces Pricing of Public Offering of Common Stock

CHICAGO, Sept. 08, 2016 (GLOBE NEWSWIRE) -- AveXis, Inc. (NASDAQ:AVXS) today announced the pricing of an underwritten public offering of 4,250,000 shares of its common stock, 3,960,145 shares of which are being offered by AveXis and 289,855 shares of which are being offered by PBM Capital Investments, LLC, an existing stockholder of AveXis, each at…

Poseida Therapeutics Establishes Scientific Advisory Board

SAN DIEGO, Sept. 08, 2016 (GLOBE NEWSWIRE) -- Poseida Therapeutics, Inc. (“Poseida”), a San Diego-based company translating best-in-class gene editing technologies into lifesaving therapeutics, announced the creation of the company's scientific advisory board with several key appointments. The scientific advisory board will provide guidance on the application of Poseida’s gene editing technology platform towards the…

AveXis Announces Proposed Public Offering of 4,000,000 Shares of Common Stock

CHICAGO, Sept. 06, 2016 (GLOBE NEWSWIRE) -- AveXis, Inc. (NASDAQ:AVXS) today announced that it intends to offer and sell, subject to market conditions, 3,566,474 shares of its common stock in an underwritten public offering. Additionally, PBM Capital Investments, LLC, an existing stockholder of AveXis, intends to offer and sell 433,526 shares in the proposed offering.…

AveXis Reports Second Quarter 2016 Financial and Operating Results and Interim Data from Ongoing Phase 1 Trial of AVXS-101 in Spinal Muscular Atrophy Type 1

-- Interim data through July 1 showed no “events” -- -- Continued motor function improvement with 25% of patients in the proposed therapeutic dose group in the normal range -- -- FDA requested Type B Meeting to discuss SMA Type 1 clinical development pathway -- -- Conference call and webcast August 11 at 4:30 p.m. EDT…

Dimension Therapeutics Reports Recent Corporate Progress and Second Quarter 2016 Financial Results

Ongoing Phase 1/2 trial DTX101 for hemophilia B clinical data expected later this year Lead IMD program, DTX301 for OTC deficiency, progressing toward IND filing Advancing broad inherited metabolic disease (IMD) portfolio with programs addressing citrullinemia type 1, phenylketonuria (PKU), and Wilson disease; activities supporting candidate selection ongoing CAMBRIDGE, Mass., Aug. 11, 2016 (GLOBE NEWSWIRE)…

Voyager Therapeutics Provides Second Quarter 2016 Investor Update

VY-AADC01 Phase 1b Study for Advanced Parkinson’s Disease On Track to Report Six-Month Safety, Motor Function, and Biomarker Data from Cohorts 1 and 2 by Year-End 2016 Pipeline Advances with Selection of Lead Clinical Candidates for Multiple Pipeline Programs by Late 2016 or Early 2017 CAMBRIDGE, Mass., Aug. 11, 2016 (GLOBE NEWSWIRE) -- Voyager Therapeutics,…

Spark Therapeutics Reports Second Quarter 2016 Financial Results and Recent Business Highlights

Updated timing of the completed BLA submission for voretigene neparvovecEarlier today, released additional positive data from the pivotal Phase 3 trial of voretigene neparvovecReported positive preliminary safety and efficacy from the Phase 1/2 clinical trial of SPK-9001 in hemophiliaPHILADELPHIA, Aug. 10, 2016 (GLOBE NEWSWIRE) -- Spark Therapeutics (NASDAQ:ONCE) today announced financial results for the quarter…

Spark Therapeutics Announces New Positive Data from Continuation of Phase 3 Trial of Voretigene Neparvovec

Of the nine crossover subjects, eight responders demonstrated the maximum improvement measurable on the primary endpoint at one year 27 of 29, or 93%, of Phase 3 subjects that received voretigene neparvovec demonstrated a gain in functional vision at one year Follow-up data show two-year durability of benefit in the Phase 3 intervention group PHILADELPHIA,…

BioMarin Announces Pricing of Public Offering of Common Stock

SAN RAFAEL, Calif., Aug. 09, 2016 (GLOBE NEWSWIRE) -- BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) announced today the pricing of an underwritten public offering of 7,500,000 shares of its common stock at a price to the public of $96.00 per share. The gross proceeds to BioMarin from this offering are expected to be approximately $720.0 million, before…

REGENXBIO Reports Second Quarter 2016 Financial Results and Recent Operational Highlights

On track to enroll first patients for Phase I/II clinical trial of RGX-501 for the treatment of homozygous familial hypercholesterolemia in the second half of 2016Received FDA Rare Pediatric Disease Designation for RGX-121 for the treatment of Mucopolysaccharidosis Type IIEntered into an exclusive license agreement with Biogen for the development of gene therapies for rare…

Voyager Therapeutics Initiates Third Cohort in Ongoing Phase 1b Clinical Trial of VY-AADC01 in Advanced Parkinson’s Disease

CAMBRIDGE, Mass., Aug. 09, 2016 (GLOBE NEWSWIRE) -- Voyager Therapeutics, Inc. (NASDAQ:VYGR), a clinical-stage gene therapy company developing life-changing treatments for severe diseases of the central nervous system (CNS), today announced dosing of the first patient in the third cohort of the ongoing Phase 1b open-label trial of VY-AADC01 in patients with advanced Parkinson’s disease…

Emerging Biotechnologies – Global Stem Cell Therapy Market Analysis, Development Trends & Key Players: MarketResearchReports.biz

Albany, NY, Aug. 09, 2016 (GLOBE NEWSWIRE) -- GlobalDatas report "PharmaSphere: Emerging Biotechnologies Stem Cell Therapy", provides strategic analysis of the global stem cells industry. It discusses key development trends, regulatory requirements in various markets, recent deals activity and industry dynamic trends, as well as describes the operations strategy of these companies. Furthermore, it includes…

Arbutus Announces Issuance of Broad New US Patent for LNP Delivery Technology

Patent Covers Lipid Particle Compositions Known to be Effective for Delivery of Nucleic Acids Including mRNA VANCOUVER, B.C. and DOYLESTOWN, Pa., Aug. 08, 2016 (GLOBE NEWSWIRE) -- Arbutus Biopharma Corporation (Nasdaq:ABUS), an industry-leading hepatitis B virus (HBV) therapeutic solutions company, today announced the issuance of a new patent, U.S. 9,404,127 (‘127 patent), that bolsters Arbutus’…

Fibrocell Announces Proposed Public Offering of Common Stock and Warrants

EXTON, Pa., Aug. 08, 2016 (GLOBE NEWSWIRE) -- Fibrocell Science, Inc. (NASDAQ:FCSC) (Fibrocell) today announced that it intends to offer and sell shares of its common stock and related warrants to purchase shares of its common stock in an underwritten public offering.  The shares of common stock and related warrants will be immediately separable and…

BioMarin Announces Public Offering of Common Stock

SAN RAFAEL, Calif., Aug. 08, 2016 (GLOBE NEWSWIRE) -- BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) announced today an offering, subject to market and other conditions, of 7,500,000 shares of its common stock in an underwritten public offering. BioMarin has also granted the underwriters a 30-day option to purchase an additional 750,000 shares of common stock. BioMarin intends…

BioMarin Announces Second Quarter 2016 Financial Results

- Second Quarter 2016 Total BioMarin Revenue Increases 20.0% Y/Y to $300.1 million- Vimizim Net Product Revenue Increases 98.1% Y/Y and Contributes $106.8 million in the Second Quarter 2016; Vimizim Full-year Revenue Guidance Increased to $340 to $360 million- Kuvan Net Product Revenue Contributes $90.2 million in the Second Quarter 2016; Kuvan Full-year Revenue Guidance…

Voyager Therapeutics to Host Second Quarter 2016 Investor Update Call

CAMBRIDGE, Mass., Aug. 04, 2016 (GLOBE NEWSWIRE) -- Voyager Therapeutics, Inc. (NASDAQ:VYGR), a clinical-stage gene therapy company developing life-changing treatments for severe diseases of the central nervous system (CNS), today announced its second quarter 2016 investor update conference call and live webcast scheduled for Thursday, August 11, 2016 at 8:30 a.m. EDT. The live call…
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