GenSight’s vision loss gene therapy again fails in phase 3

Questex LLC | February 04, 2019

A phase 3 trial of GenSight Biologics’ Leber hereditary optic neuropathy (LHON) gene therapy has missed its primary endpoint. The AAV gene therapy was no better than placebo at improving vision at 48 weeks, leading GenSight to look to future updates to salvage the study. France’s GenSight designed GS010 to improve the vision of patients with a particular mutation in the mitochondrial ND4 gene and moved the gene therapy into a pair of phase 3 trials in 2016. One trial enrolled patients who had suffered vision loss for 6 to 12 months. The other recruited people whose vision loss began less than six months ago. Both trials missed their primary endpoints. The latest clinical setback involves LHON patients with six months or less of vision loss enrolled in the RESCUE trial. As in the other study, GenSight set out to link GS010 to a 15-letter improvement over placebo on a vision test. Each subject received GS010 in one eye and a sham injection in the other.

Spotlight

As we grow and develop, very specific chemical reactions activate and deactivate parts of our genome at strategic times and in specific locations, essentially turning our genes on and off. Epigenetics is the study of these processes and the factors that influence them.So why care about epigenetics if it is only in your cells? What could you possibly do to affect your DNA? It turns out that what you eat, where you live, how you react, when you sleep, if you exercise, and even how you age can affect epigenetics.

Spotlight

As we grow and develop, very specific chemical reactions activate and deactivate parts of our genome at strategic times and in specific locations, essentially turning our genes on and off. Epigenetics is the study of these processes and the factors that influence them.So why care about epigenetics if it is only in your cells? What could you possibly do to affect your DNA? It turns out that what you eat, where you live, how you react, when you sleep, if you exercise, and even how you age can affect epigenetics.

Related News

INDUSTRY OUTLOOK

Andelyn Biosciences Selects TetraScience as a Strategic Partner for Innovation in Therapeutics Development and Manufacturing

Andelyn Biosciences | August 26, 2021

TetraScience, the R&D Data Cloud company, announced today that Andelyn Biosciences, Inc. has selected the Tetra Data Platform (TDP) to build a cloud-native development and manufacturing service for its customers. As part of an Andelyn Biosciences initiative to create a Connected Plant that harmonizes data from building and facilities, process and manufacturing, and lab instrument sources, TDP will enable better compliance, easier analysis, faster decisions, and more agile operations. Additionally, Andelyn customers will benefit from secure, convenient access to harmonized data that is prepared for analytics and visualization. As a Contract Development and Manufacturing Organization (CDMO) for cell and gene therapies, Andelyn must remain GMP-compliant and increase efficiency while leading the industry in the innovative services they provide to customers. By launching a Connected Plant, Andelyn will gain autonomous operation, predictive capabilities, improved process monitoring and control, and increased data integrity. "Andelyn has been a pioneer in gene therapy with a 15-year history accelerating the development and manufacturing of innovative therapies to bring more treatments to more patients," said Bryan J. Holmes, Vice President, Information Technology at Andelyn Biosciences. "Together with TetraScience, we're transforming how we work with our clients, utilizing fully-digitized, cloud-native services to help connect all the plant and lab data created during complex development and manufacturing cycles." Andelyn chose TDP for its unique ability to harmonize data from building & facilities systems, lab instruments, and process & manufacturing equipment. By replatforming the data to a future-proof managed cloud environment, Andelyn will benefit from enhanced GMP compliance and data integrity, improved time-to-value, and lower TCO. Additionally, with an open data cloud platform powered by TDP and the Tetra Partner Network, Andelyn will be able to leverage their preferred tooling for analytics while supporting their customers' data consumption needs. "We're excited to partner with Andelyn Biosciences on their mission-critical data cloud initiative and accelerate their journey towards becoming a digital company," stated Chad Garrett, TetraScience CRO. "As an innovator in providing cloud-native development and manufacturing services to its customers, Andelyn Biosciences recognizes that harmonizing scientific data in the cloud and making it easily accessible to clients will transform the way CDMOs exchange data with clients." Andelyn's initial deployment of TDP is focused on acquiring and harmonizing historical data across key sources while enabling enhanced data search capabilities. Moving forward, Andelyn expects to complete the rollout of the Connected Plant with additional data sources and instruments, and ultimately launch a groundbreaking digital CDMO experience for their customers. About Andelyn Biosciences, Inc. Andelyn Biosciences is a pioneering gene therapy company born out of Nationwide Children's Hospital, where the first FDA-approved systemic gene therapy was discovered. Andelyn is named in honor of two gene therapy pioneers who participated in pivotal Phase I clinical trials at Nationwide Children's Hospital 8-year-old Andrew and 8-week-old Evelyn. Bringing together more than 15 years of experience in AAV vector manufacturing, Andelyn is a full-spectrum Contract Development Manufacturing Organization (CDMO) that helps pioneer solutions that turn hope into reality. Our mission is to accelerate the development and manufacturing of innovative therapies to bring more treatments to more patients. We accomplish this by bringing scalability, capacity, efficiency and expert resources to manufacturing matrices, all to advance the development of viral vector-based therapeutics. Our capabilities include viral vector process and analytical development, small to large scale GMP manufacturing, fill/finish and QC/QA release testing through Phase III with plans to launch commercial services in 2022. Andelyn also offers plasmid manufacturing services, formulation development services and regulatory support.

Read More

CELL AND GENE THERAPY

Amyris Partners with Inscripta to Enhance Development of Sustainable Ingredients Using the Onyx™ Genome Engineering Platform

Amyris | October 06, 2021

Amyris, Inc. (Nasdaq: AMRS), a leading synthetic biotechnology company active in the Clean Health and Beauty markets through its consumer brands, and a top supplier of sustainable and natural ingredients, today announced that Amyris has licensed the Onyx genome engineering platform from Inscripta, a leading gene editing technology company. Amyris and Inscripta will also explore joint research and development opportunities to expand the Onyx platform functionality. Amyris' product development and formulation team uses a proprietary Lab-to-Market™ operating system to develop and scale a growing portfolio of sustainable ingredients. The Onyx platform automates benchtop biofoundry activity and will bring greater genetic diversity and value to Amyris' ingredient development pipeline, complementing Amyris' existing Lab-to-Market operating system with the goal of improving efficiency and reducing timelines for the development of future molecules. To date, Amyris has successfully commercialized 13 sustainable ingredients, which are formulated in over 20,000 products and used by over 300 million consumers, demonstrating the growing demand for sustainable products with clean and effective ingredients. Automated, high-throughput gene editing is revolutionizing the writing of genomes the way next-generation sequencing transformed the reading of genomes. Inscripta is the first company to deliver an integrated and intuitive benchtop platform that will expand access to scalable, robust genome engineering and help scientists develop solutions to some of today's most pressing challenges. "Amyris has shown the world how new products can be made more sustainable through biology. Their team has high proficiency in utilizing cutting-edge technology, and we are excited they will be pioneering the use of our platform," said Sri Kosaraju, President and CEO of Inscripta. "We have great regard for Amyris' mission, and we are committed to seeing the Onyx platform become a substantial contributor to new clean chemistry products in the future." "The Onyx platform offers significant potential for generating greater genetic diversity in our projects, which we expect to lead to more efficient product innovation," said Sunil Chandran, Senior Vice President of Research and Development at Amyris. "Inscripta's platform seamlessly integrates with our own and opens up new experimentation avenues for our scientists to continue bringing unique bio-based products to customers. We pride ourselves on continuous innovation and expect Onyx to help us expand our pipeline, while achieving lower costs and reducing time to market." For more information about Amyris visit amyris.com and to learn about Onyx, visit www.inscripta.com/products. About Inscripta Inscripta is a life science technology company enabling scientists to solve some of today's most pressing challenges with the first benchtop system for genome editing. The company's automated Onyx platform, consisting of an instrument, consumables, assays, and software, makes CRISPR-based genome engineering accessible to any research lab. Inscripta supports its customers around the world from facilities in Boulder, Colorado; San Diego and Pleasanton, California; and Copenhagen, Denmark. To learn more, visit Inscripta.com and follow @InscriptaInc. About Amyris Amyris (Nasdaq: AMRS) is a science and technology leader in the research, development and production of sustainable ingredients for the Clean Health & Beauty and Flavors & Fragrances markets. Amyris uses an impressive array of exclusive technologies, including state-of-the-art machine learning, robotics and artificial intelligence. Our ingredients are included in over 20,000 products from the world's top brands, reaching more than 300 million consumers. Amyris is proud to own and operate a family of consumer brands - all built around its No Compromise® promise of clean ingredients: Biossanceâ clean beauty skincare, Pipetteâ clean baby skincare, Purecane™, a zero-calorie sweetener naturally derived from sugarcane, Terasanaâ clean skincare treatment, Costa Brazil luxury skincare, OLIKA hygiene and wellness, Rose Inc.™ clean color cosmetics and JVN™ clean haircare.

Read More

Micreos Human Health Enrolles World's First Endolysin-drug as a Therapy for Atopic Dermatitis

Micreos | September 22, 2020

Dutch biotech company Micreos Human Health has enrolled the first patients in a Phase I/IIa, randomized, double-blind, placebo-controlled, parallel treated dose-ranging study to assess the safety and efficacy of XZ.700 in patients with mild to moderate atopic dermatitis. The study marks the world's first evaluation of a pharmaceutical endolysin for topical use in humans. Endolysins are highly specific enzymes that cut the bacterial cell wall, rapidly killing only the target bacteria, regardless of antibiotic resistance, while preserving the skin microbiome. XZ.700 specifically targets Staphylococcus aureus (S. aureus), a bacterium that is considered to be a causative and aggravating trigger for atopic dermatitis, and viewed as an independent cause of itch, irritation and infection. "The targeted removal of one particular bacterial species, S. aureus, from the skin microbiome, while preserving the beneficial ones is a fundamental new way to treat atopic dermatitis," says dermatologist Dr. Peter Lio, Scientific Advisor for Micreos and Scientific Advisory Board member for the National Eczema Association. XZ.700 is one of several endolysins Micreos has in its portfolio. A structurally similar endolysin, SA.100, is used in the company's over the counter (OTC) products for inflammatory skin disorders (e.g. acne and rosacea), found in the Gladskin range, which have been on the market since 2013. Data obtained in earlier Gladskin studies and feedback from doctors and thousands of customers support the pharmaceutical development program: "I am very encouraged by my experience with Gladskin and I am awaiting the findings with great anticipation." In this study, conducted in The Netherlands, XZ.700 will be tested for its safety as well as pharmacodynamics and efficacy in 48 patients with atopic dermatitis. They will be treated for 14 days with a cream containing XZ.700 at three different concentrations or placebo. Micreos expects to finish the study and report the results towards the end of 2021.

Read More