Tag Archives: AGTC

AGTC Treats First Patient of Second Cohort in Phase 1/2 Clinical XLRP Study; Earns $10 Million Enrollment Milestone (BioFlorida)

Applied Genetic Technologies Corporation, a biotechnology company conducting human clinical trials of adeno-associated virus (AAV)-based gene therapies for the treatment of rare diseases, announced that it has enrolled the first patient of the second cohort in the company’s Phase 1/2 clinical trial evaluating the safety and efficacy of an investigational AAV-based gene therapy for the treatment of X-linked retinitis pigmentosa (XLRP). Under the terms of its collaboration with Biogen, AGTC will receive a milestone payment of $10 million. AGTC is a UF Innovate | Sid Martin Biotech company that has licensed UF technology.

“We are pleased to announce this important milestone under our collaboration with Biogen and remain on track to complete the dose escalation portion of the trial in the first quarter of 2019,” said Sue Washer, President and CEO of AGTC.

First Patient Dosed in X-Linked Retinitis Pigmentosa (XLRP) Treatment (RareDR)

UF startup Applied Genetic Technologies Corporation announced that the first patient was dosed in its Phase 1/2 clinical trial evaluating the safety and efficacy of AGTC-501 for the treatment of X-linked retinitis pigmentosa (XLRP).

The investigational adeno-associated virus (AAV-based) gene therapy, (rAAV2tYF-GRK1-RPGR), is a novel recombinant AAV vector expressing a human RPGR gene (rAAV2tYF-GRK1-RPGR) in patients with XLRP.

XLRP is an inherited condition that affects young boys and men. It causes the development of night blindness and eventually leads to legal blindness by patients’ early forties.

AGTC Announces Completion of Enrollment of Phase 1/2 Clinical Study of Investigational Gene Therapy in Patients with X-Linked Retinoschisis (Globe Newswire)

UF startup and UF Innovate | Sid Martin Biotech alumni company Applied Genetic Technologies Corporation announced the completion of enrollment in a clinical study of the company’s gene therapy product candidate, in collaboration with Biogen, for the treatment of x-linked retinoschisis (XLRS). AGTC is a biotechnology company conducting human clinical trials of adeno-associated virus (AAV)-based gene therapies for the treatment of rare diseases. This multicenter study is designed to evaluate the collaboration’s AAV vector expressing retinoschisin (rAAV2tYF-CB-hRS1) in patients with XLRS caused by mutations in the RS1 gene. Topline data are anticipated by Q4 2018 with the final analysis at the twelve-month time point.

“There are currently no FDA approved treatment options for XLRS, a leading cause of macular degeneration in young men,” said Sue Washer, president and CEO of AGTC. “The completion of enrollment in this AAV trial represents another significant achievement in our gene therapy clinical development program- a milestone that may improve the lives of individuals affected by XLRS.”

AGTC Announces Sue Washer, CEO, Elected to Biotechnology Innovation Organization’s Board of Directors (GlobeNewswire)

Applied Genetic Technologies Corporation, a UF biotechnology company conducting human clinical trials of adeno-associated virus (AAV)-based gene therapies for the treatment of rare diseases, announced the election of Sue Washer, President and Chief Executive Officer, to the Biotechnology Innovation Organization (BIO)’s Board of Directors.

“For more than two decades, BIO has been a champion of the rapidly expanding biotechnology industry and I am honored to be elected to the organization’s Board of Directors,” said Ms. Washer. “My primary focus will be to enhance BIO’s position as a critical resource to fledgling biotechnology companies, ensuring they have access to the proper tools and information to reach commercialization. In addition, I am committed to working closely with federal, state and local governmental representatives to advance the development of transformative therapies addressing serious unmet medical needs.”

ACTC Files Investigational New Drug Application for the Treatment of X-Linked Retinitis Pigmentosa (GlobeNewswire)

Applied Genetic Technologies Corporation, a UF biotechnology company conducting human clinical trials of adeno-associated virus (AAV)-based gene therapies for the treatment of rare diseases, announced that it has filed an Investigational New Drug application (IND) with the U.S. Food and Drug Administration to conduct a Phase 1/2 clinical trial of the company’s gene therapy product candidate for the treatment of X-linked retinitis pigmentosa (XLRP) caused by mutations in the RPGR gene.

XLRP is an inherited condition that causes progressive vision loss, beginning with night blindness in young boys followed by progressive constriction of the field of vision. Affected men progress to legal blindness by their early forties. Preclinical data indicated that treatment with an investigational gene therapy product slowed the loss of visual function in canines with XLRP caused by mutations in the RPGR gene. XLRP and X-linked retinoschisis (XLRS) are the two lead development programs within AGTC’s collaboration and license agreement with Biogen, Inc., to develop gene-based therapies for multiple ophthalmic diseases.

AGTC Announces Publishing of Research Supporting Clinical Development of Gene-Based Therapy for Treatment for X-Linked Retinitis Pigmentosa (StreetInsider)

Researchers from the University of Pennsylvania announced the publication of preclinical proof of concept data supporting the clinical development of a gene-based therapy for the treatment of X-linked retinitis pigmentosa (XLRP), one of the most common inherited retinal disorders, which causes progressive vision loss in boys and young men.

The investigational gene therapy was developed by UF startup Applied Genetic Technologies Corporation (AGTC), a biotechnology company conducting human clinical trials of adeno-associated virus (AAV)-based gene therapies for the treatment of rare diseases. AGTC is developing a gene-based therapy for XLRP in collaboration with Biogen.

AGTC Appoints William Sullivan as CFO, Andrew Ashe Named General Counsel (Nasdaq GlobeNewswire)

UF startup Applied Genetic Technologies Corporation (AGTC), a biotechnology company conducting human clinical trials of adeno-associated virus (AAV)-based gene therapies for the treatment of rare diseases, announced the appointments of William Sullivan to the position of Chief Financial Officer and Andrew Ashe to the position of General Counsel.  Larry Bullock, who has served as Chief Financial Officer, is retiring.

AGTC Announces U.S. FDA Orphan Drug Designation for Gene Therapy to Treat X-Linked Retinitis Pigmentosa (Globe NewsWire)

Applied Genetic Technologies Corporation, a biotechnology company conducting human clinical trials of adeno-associated virus (AAV)-based gene therapies for the treatment of rare diseases, announced that the U.S. Food and Drug Administration (FDA) has granted an orphan drug designation for its gene therapy product candidate for the treatment of X-linked retinitis pigmentosa (XLRP) caused by mutations in the RPGR gene. In June 2016 the company announced that the European Commission (EC) granted orphan medicinal product designation for the same indication.

XLRP is an inherited condition that causes progressive vision loss, beginning with night blindness in young boys followed by progressive constriction of the field of vision. Affected men become legally blind at an average of about 45 years of age. The most common form of XLRP is caused by mutations in the RPGR gene. Preclinical data indicate that treatment with a gene therapy product slowed the loss of visual function in a canine model of XLRP due to mutations in the RPGR gene. XLRP and X-linked retinoschisis (XLRS) are the two development programs within AGTC’s collaboration and license agreement with Biogen Inc., to develop gene-based therapies for multiple ophthalmic diseases.

Preclinical Data Demonstrates Efficacy of AGTC-402 Therapy for Achromatopsia (EIN Newsdesk)

UF startup Applied Genetic Technologies Corporation, a biotechnology company conducting human clinical trials of adeno-associated virus (AAV)-based gene therapies for the treatment of rare diseases, announced the publication of IND-enabling preclinical data demonstrating the safety and efficacy of AGTC-402 in the treatment of achromatopsia (ACHM) due to mutations in the CNGA3 gene.

The study, “Safety and Efficacy Evaluation of rAAV2tYF-PR.1-hCNGA3 Vector Delivered by Subretinal Injection in CNGA3 Mutant Achromatopsia Sheep” appears in the June issue of Human Gene Therapy Clinical Development and was led by researchers at the Volcani Center and Hebrew University in Israel. The gene-based therapy used in this study, AGTC-402, utilizes AGTC’s proprietary AAV technology to deliver a functional copy of the human CNGA3 gene.

Why is Gainesville Florida’s Biotech Hub? (IndustryWeek)

Moving from an economy that relies heavily on tourism to one that has a large stake in biotech is a big leap. And it calls for a long-term, well developed strategy. That’s what Governor Jeb Bush was after back in 2003. He began by using federal stimulus dollars as bait, luring the Scripps Research Institute to Florida. He next urged the state legislature in 2006 to create an Innovation Investment Fund to attract world-class research and development. His efforts worked, eventually allowing state universities and their research partners to leverage public and private dollars to commercialization of emerging technologies.

All of this success has translated into financial gains for cities across the state, and Gainesville is one city that has seen its biotech economy grow by leaps and bounds.

“Greater Gainesville has evolved into a region fueled by research, business and a smart, young and creative workforce that both thrives on, and is a catalyst for innovation,” said Susan Davenport, CEO of Gainesville Chamber of Commerce.