2021 PRESENTING COMPANIES
February 18th, 2021 at the following time:
1:00PM - 4:30PM Eastern Time
Adverum Biotechnologies
Adverum is a clinical-stage gene therapy company targeting unmet medical need in ocular and rare diseases. We develop gene therapy product candidates designed to provide durable efficacy by inducing sustained expression of a therapeutic protein. Our core capabilities include clinical development, novel vector discovery, and in-house manufacturing expertise, specifically in scalable process development, assay development, and current Good Manufacturing Practices (cGMP) quality control. Our team has extensive clinical development experience and is laser-focused on advancing novel gene therapies for patients.
AGTC
AGTC is a clinical-stage biotechnology company that uses a proprietary gene therapy platform to develop transformational genetic therapies for patients suffering from rare and debilitating diseases. Its initial focus is in the field of ophthalmology, where it has active clinical trials in X-linked retinitis pigmentosa (XLRP) and achromatopsia (ACHM CNGB3 & ACHM CNGA3). In addition to its clinical trials, AGTC has preclinical programs in optogenetics, other ophthalmology, CNS and otology indications. The optogenetics program is being developed in collaboration with Bionic Sight and the otology program is partnered with Otonomy. In addition to its product pipeline, AGTC has a significant intellectual property portfolio and extensive expertise in the design of gene therapy products including capsids, promoters and expression cassettes, as well as expertise in the formulation, manufacture and physical delivery of gene therapy products.
Eyevensys
Eyevensys is a France-based, VC-backed biotech company, also incorporated in USA. Eyevensys is developing a novel non-viral gene therapy for treatment of ocular diseases. The technology is comprised of a proprietary device that delivers plasmid DNA to the ciliary muscle. After electro-transfection, the muscle cells sustainably produce the therapeutic proteins that the plasmid encodes.
The company has successfully completed part 1 of it's clinical safety study in France and the UK, enrolling 9 patients with late stage non-infectious uveitis. Study is being expanded in less severe patients - An IND is being filed in USA in order to validate technology in a Phase 2 Clinical study -non-infectious uveitis .
Parallel programs are pursued with different proteins-one for treatment of retinitis pigmentosa, one for Wet AMD.
GenSight Biologics
GenSight Biologics S.A. is a clinical-stage biopharma company focused on developing and commercializing innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders. GenSight Biologics’ pipeline leverages two core technology platforms, the Mitochondrial Targeting Sequence (MTS) and optogenetics, to help preserve or restore vision in patients suffering from blinding retinal diseases. GenSight Biologics’ lead product candidate, LUMEVOQ® (GS010; lenadogene nolparvovec), has been submitted for marketing approval in Europe for the treatment of Leber Hereditary Optic Neuropathy (LHON), a rare mitochondrial disease affecting primarily teens and young adults that leads to irreversible blindness. Using its gene therapy-based approach, GenSight Biologics’ product candidates are designed to be administered in a single treatment to each eye by intravitreal injection to offer patients a sustainable functional visual recovery.
Iveric Bio
Iveric Bio is a science-driven biopharmaceutical company focused on the discovery and development of novel treatment options for retinal diseases with significant unmet medical needs. The Company is currently developing both therapeutic product candidates for age-related retinal diseases and gene therapy product candidates for orphan inherited retinal diseases. Vision is Our Mission. For more information on the Company, please visit www.ivericbio.com.
Ocugen
Ocugen, Inc. is a biopharmaceutical company focused on discovering, developing, and commercializing transformative therapies to cure blindness diseases and developing a vaccine to fight COVID-19. Our breakthrough modifier gene therapy platform has the potential to treat multiple retinal diseases with one drug – “one to many” and our novel biologic product candidate aims to offer better therapy to patients with underserved diseases such as wet age-related macular degeneration, diabetic macular edema, and diabetic retinopathy. Ocugen is also co-developing Bharat Biotech’s COVAXIN™ vaccine candidate for COVID-19 in the US market. For more information, please visit www.ocugen.com.
REGENXBIO
REGENXBIO Inc. is a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy. Our gene therapy product candidates are designed to deliver functional genes to address genetic defects in cells, enabling the production of therapeutic proteins or antibodies that are intended to impact disease. Through a single administration, gene therapy could potentially alter the course of disease significantly and deliver improved patient outcomes with long-lasting effects. We have developed a broad pipeline of gene therapy programs using our NAV® Technology Platform to address genetic diseases through two modalities: AAV-mediated antibody delivery and monogenic gene replacement. We believe this platform forms a strong foundation for our current programs and with our ongoing research and development, we expect to continue to expand the platform.
SparingVision
SparingVision is a genomic medicines company, translating pioneering science into vision saving treatments. Founded to advance over 20 years of world-leading ophthalmic research from its scientific founders at the Paris Vision Institute, SparingVision is leading a step shift in how ocular diseases are treated, moving beyond single gene correction therapies. At the heart of this is SPVN06, a gene independent treatment for retinitis pigmentosa (RP), the most common inherited retinal disease affecting two million people worldwide. SPVN06 could form the basis of a suite of new sight saving treatments as it could be applicable to many other retinal diseases, regardless of genetic cause.
The Company is supported by a strong, internationally renowned team who aim to harness the potential of genomic medicine to deliver new treatments to all ocular disease patients as quickly as possible. SparingVision has raised €60 million to date and its investors include 4BIO Capital, Bpifrance, Foundation Fighting Blindness (US), Fondation Voir & Entendre, UPMC Enterprises, Jeito Capital and Ysios Capital. For more information, please visit www.sparingvision.com.