Roche Buys Ex-U.S. Rights to Sarepta DMD Gene Therapy Candidate SRP-9001 for Up-to-$2.85B+

Genetic Engineering and Biotechnology News | December 23, 2019

Roche has acquired exclusive commercial rights outside the U.S. to Sarepta Therapeutics’ lead gene therapy pipeline candidate SRP-9001 (AAVrh74.MHCK7.micro-dystrophin) for Duchenne muscular dystrophy (DMD), through a licensing agreement that could generate more than $2.85 billion for Sarepta, the companies said today. SRP-9001, currently in Phase II clinical development, is designed to deliver the micro-dystrophin-encoding gene directly to the muscle tissue for the targeted production of the micro-dystrophin protein. Sarepta is recruiting patients for the two-part Phase II SRP-9001-102 trial (NCT03769116), a 40-patient study designed to assess the safety and efficacy of SRP-9001 in a 48-week randomized, double-blinded, placebo-controlled period (Part 1), and a 96-week, double-blinded extension period (Part 2). The study has an estimated primary completion date of October 10, 2022.

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How do you determine if your inventory is automation friendly? Do you want to automate your biobank? Chances are if you didn't take into account automation in your biobank, there are some serious challenges ahead for you. In this eBook, we focus on 10 considerations every biobank should think about.

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Sestina Bio | October 17, 2020

Sestina Bio, a new company creating a platform for automated synthetic biology, today announced that it has launched with seed funding from Foresite Capital, General Inception and OMX Ventures. The funds will be used to further the development of its digital cellular engineering and phenotyping platforms and support initial product development. The firm is the first company to launch from incubator Foresite Labs. Practitioners of synthetic biology aim to engineer novel living organisms -- usually yeast and bacteria -- that produce new materials, fine and bulk chemicals, and pharmaceuticals, or which act as sensors that report on their environment. By combining new instruments for single cell genome editing, tools for single cell phenotyping, and closed-loop, machine learning-driven design and control of experiments, Sestina is developing the next generation of the automated synthetic biology toolkit. Sestina will harness these tools to rapidly and deterministically engineer new biological capabilities and produce novel products across several verticals.

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Felix Biotechnology Announces Initiation of CYPHY Phase 1/2 study at Yale for Lead Asset

Felix Biotechnology | January 08, 2021

Felix Biotechnology reported the inception of CYPHY, a Phase 1/2 examiner started single focus preliminary at Yale University for focused phage treatment YPT-01 in the therapy of constant P. aeruginosa diseases in cystic fibrosis. This twofold visually impaired, fake treatment controlled study (NCT 04684641) will evaluate the wellbeing and adequacy of YPT-01 added to standard antimicrobial treatment in 36 patients. CYPHY will likewise evaluate the capacity of YPT-01 to lessen the harmfulness and anti-infection obstruction of P. aeruginosa, improving patient results and re-empowering utilization of conventional anti-infection agents against multi-drug safe strains. The lead specialist for this investigation, Dr. Jon Koff, Associate Professor and Director of Yale's Adult Cystic Fibrosis Program, is supported by an academic grant from the Cystic Fibrosis Foundation. "This is a fantastic opportunity to show how effective phage therapy can be when deployed in an evolutionary framework. We know that pathogens evolve resistance to any antibiotic or therapy we use, so our approach turns that to our advantage," said Dr. Paul Turner, Professor of Ecology and Evolutionary Biology, co-inventor of YPT-01, and co-founder of Felix Biotechnology. "By targeting phage to mechanisms of virulence, we ensure that if pathogens evolve resistance to phage, they lose traits that make them effective pathogens, putting them in an evolutionary Catch-22."

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Ridgeback Biotherapeutics LP Announces the U.S. Food and Drug Administration Approvel of EbangaTM for Ebola

Ebanga | December 29, 2020

Ridgeback Biotherapeutics LP ("Ridgeback"), a biotechnology organization experienced in antiviral drug improvement, reported today that the U.S. Food and Drug Administration ("FDA") approved EbangaTM for the treatment of Ebola. Ebanga is presently affirmed for treatment of disease brought about by Zaire ebolavirus in grown-up and pediatric patients. Ebanga is the only FDA endorsed, single infusion Ebola treatment which is accessible in a lyophilized structure. Wendy Holman, CEO and co-founder of Ridgeback, said: “I speak on behalf of the entire Ridgeback Bio team when I say thank you to all of the dedicated and thoughtful groups who helped contribute to this historic and important product approval. Ridgeback is grateful to NIAID’s Vaccine Research Center (VRC) and the Democratic Republic of the Congo’s (DRC) Institut National pour la Recherche Biomédicale (INRB). Specifically, the brilliance of three individuals needs to be acknowledged -- Dr. Nancy Sullivan (VRC), Dr. John Mascola (VRC) and Professor Jean-Jacques Muyembe-Tamfum (INRB). It was their mission to create a lyophilized single-use Ebola treatment, and in partnership with the team at Ridgeback, that vision became a reality. We would also like to thank FDA leadership and the Ebanga review team at FDA’s Division of Antivirals (DAV) for their focused and thorough review of this product. Finally, the true heroes are the patients and their families who allowed themselves to be carried away from their homes – not knowing if they would ever return – in order to enroll in a randomized controlled trial at the Ebola treatment centers during the 2018-2020 Ebola outbreak in the DRC. Your trust and belief in science, innovation and the people caring for you has transformed Ebola forever. Thanks to you, Ebola is now a treatable disease.”

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