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FDA Grants RMAT To Sangamo Therapeutics’ Gene Therapy for Hemophilia A (SB-525)

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The FDA has granted RMAT designation for Sangamo Therapeutics’ SB-525, an investigational gene therapy being developed in collaboration with Pfizer to treat severe hemophilia A. SB-525 is the first hemophilia A gene therapy to receive RMAT designation.

According to Sangamo Therapeutics:

“We are encouraged by the initial clinical data suggesting safety, tolerability, and efficacy of SB-525 and are beginning preparations, including manufacturing, to potentially advance into a registrational study. We are also encouraged by our interactions with regulators and by the FDA’s recent RMAT designation,” said Seng Cheng, Senior Vice President and Chief Scientific Officer of Pfizer’s Rare Diseases Research Unit. “If FVIII levels are sustained, and patients continue to have no bleeding episodes and remain off factor replacement therapy, we believe that this gene therapy may potentially represent a transformative treatment paradigm for severe hemophilia A.”

FDA Approved RMAT Designations

To date, 30 RMAT designations have been publicly announced. The most recent additions were Krystal Biotech’s RMAT designation for KB103 on June 24, 2019 and Sangamo Therapeutics’ RMAT designation for SB-525 on July 5, 2019.

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What is an RMAT and Who Has One? (30 Announced / 40 Granted)

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As of today, the list of publicly announced RMAT’s has risen to 30. However, the U.S. FDA states it has received 108 total requests for RMAT designations and granted 40.

RMAT stands for “Regenerative Medicine Advanced Therapy” designation. Sponsors of cell and gene therapies are eligible to obtain an RMAT designation from the U.S. FDA if their product is intended to treat serious or life-threatening diseases and there is preliminary clinical evidence that it can address unmet medical needs.

In this article:

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Century Therapeutics Launches With $250 Million for Allogeneic iPSC Platform

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  • Created by Versant Ventures and partnered with Fujifilm to develop iPSC-derived adaptive and innate immune effector cell therapies

  • Leaps by Bayer leads investment round with USD 215 million commitment to develop next-generation immune oncology treatments

July 01, 2019, LEVERKUSEN, Germany & PHILADELPHIA & TOKYO–Century Therapeutics, which was created by Versant Ventures as part of the firm’s core strategy to launch innovative biotech companies, emerged from stealth mode today with USD 250 million in financing commitments from Bayer, Versant and Fujifilm Cellular Dynamics Inc. (FCDI). The proceeds will enable Century to advance multiple programs into the clinic for hematologic and solid malignancies. [Read more…]

Cellara, LLC, releases CultureTrax, Productivity Software Designed for the Unique Complexities of Stem Cell Research

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Productivity software enables tracking, sharing and collaborating around stem cell research to accelerate scientific discoveries and breakthroughs.

CultureTraxMadison, WI, June 3, 2019 — Cellara, LLC, a software-as-a-service company, announced the commercial release of CultureTrax software V2.0. It is the first productivity software designed to meet the unique needs of stem cell scientists.

Stem cells are a core technology for basic biological and medical research as well as the foundation of rapidly emerging cell-based therapies and disease models for drug development. Discoveries are built on stem cell scientists’ ability to communicate quickly and accurately with one another and reproduce their own work or the work of others. Studies indicate more than 50% of published pre-clinical biology research cannot be easily reproduced (Freedman, et al, 2015).

Outdated methods for documenting experimental details and sharing protocols using static documents and paper lab notebooks are a major source of the problem. Although many labs are adopting electronic lab notebooks, these have not solved the complex cell line tracking and reproducibility challenges unique to stem cell research.

“Our vision is to accelerate scientific discoveries and breakthrough therapies by providing advanced cell culture software,” says CEO Scott Fulton. “CultureTrax software enables you to quickly and easily plan and fully document your cell culture and analytical work. The software also provides a repository for detailed, current protocols and recipes, structured to drive accurate execution in the lab and rapid, comprehensive recording of results.”

Professor Rebekah Gundry of Medical College of Wisconsin has been using CultureTrax software in her lab for two years, and states, “CultureTrax provides immediate, paperless access to protocols, recipes and the experimental plan which minimizes human error and saves us time.”

CultureTrax is a secure web-based application, so you can access your work from anywhere through a browser on your PC or tablet. CultureTrax enables you to easily share both your experimental workload, your protocols and your detailed results with colleagues. This improves the ability of scientists to replicate work and enhance efficiency and progress in the field of stem cell research. For more information, visit www.culturetrax.com.

About Cellara

Cellara is a software-as-a-service company that has developed CultureTrax, a powerful stem cell productivity platform capable of managing the complex needs of stem cell research. It is a Wisconsin-based company headquartered in Madison, a world-class center of the most advanced cell culture science, life science tools industry and information technology. CultureTrax creates a common digital language to empower cell culture scientists to realize the full potential of their research and collaborate more effectively.

Do you have questions about Cellara or its CultureTrax software V2.0? Ask them in the comments below.

Krystal Biotech Receives RMAT Designation from FDA for Gene Therapy, KB103

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On June 24, 2019, Krystal Biotech (NASDAQ:KRYS) became the latest regenerative medicine company to announce that it has received an RMAT designation granted from the FDA for its gene therapy product, KB103. The FDA’s decision was based on positive results from the company’s Phase 2 placebo-controlled clinical trial of KB103 and an update on the results from its Phase 1 study. These studies are named GEM-1 and GEM-2.

The same day, the company announced it had initiated an underwritten public offering of $100.0 million of its common stock. Among other activities, Krystal Biotech intends to use these proceeds to advance KB103 into and through a Phase 3 clinical trial.

Krystsal Biotech’s RMAT Designation

RMAT stands for “Regenerative Medicine Advanced Therapy” designation. Sponsors of cell and gene therapies are eligible to obtain an RMAT designation from the U.S. FDA if their product is intended to treat serious or life-threatening diseases and there is preliminary clinical evidence that it can address unmet medical needs.

The RMAT designation is well-matched to Krystal Biotech’s product development focus, because it is a gene therapy company focused on commercializing treatments for patients suffering from dermatological diseases.

KB103 is currently being studied for its wound closure capacity in patients with severe generalized recessive dystrophic epidermolysis bullosa (“RDEB”).

As stated by the company:

“KB103’s RMAT designation follows our recent announcement of the Priority Medicines, or PRIME designation received from the EMA, and we look forward to collaborating closely with the EMA and initiating a clinical trial in the EU in the upcoming months. The RMAT pathway is analogous to the Breakthrough Therapy designation designed for traditional drug candidates and medical devices and was specifically created by the U.S. Congress in 2016 to get important new cell therapy and gene therapy products to the patient earlier.

Just like the Breakthrough designation, it allows companies developing regenerative medicine therapies to interact with the FDA more frequently in the clinical testing process, and RMAT-designated products may be eligible for priority review and accelerated approval.”

Krystal Biotech

Headquartered in Pittsburgh, PA, Krystal Biotech is planning a pivotal Phase 3 trial for KB103 planned to start before 2020.

To learn more about Krystal Biotech, view this company presentation by Suma Krishnan, Founder and Chief Operating Officer  (COO).

Seeking a searchable, sortable Excel database of RMATdesignations you can use on-the-go? Access one here.

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