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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.

FDA Awards Latest RMAT to Audentes Therapeutics for Gene Therapy Product AT132

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On August 21, 2018, Audentes Therapeutics, Inc. (Nasdaq: BOLD ) announced that the U.S. FDA granted Regenerative Medicine Advanced Therapy (RMAT) designation to AT132, its treatment for X-linked Myotubular Myopathy (XLMTM).

Audentes is a biotechnology company that develops gene therapy products for patients for rare diseases.

Audentes has pursued a number of advanced regulatory pathways for AT132, including:

  • Regenerative Medicine Advanced Therapy (RMAT), Rare Pediatric Disease, Fast Track and Orphan Drug designations by the FDA
  • Priority Medicines (PRIME) and Orphan Drug designations by the European Medicines Agency (EMA)

[Read more…]

Lonza Opens World’s Largest Dedicated Cell-and-Gene-Therapy Manufacturing Facility

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  • First-of-its-kind, state-of-the-art manufacturing facility with capacity to produce treatment for thousands of patients suffering from rare genetic disorders or life-threatening diseases

  • With 300,000 square feet (27,870 square meters), it is the largest dedicated cell-and-gene-therapy manufacturing facility in the world

  • Facility to reach 200 full-time staff by end of 2018 and continue to recruit high-value positions as market demand increases

[Read more…]

BioLife Solutions Customer Kite Pharma Receives FDA Approval for Yescarta™ CAR T-Cell Therapy

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Each Yescarta Dose is Frozen in Proprietary CryoStor® Freeze Media to Maintain CAR T-Cell Viability and Enable Worldwide Distribution

BOTHELL, Washington — October 19, 2017 —BioLife Solutions, Inc. (NASDAQ: BLFS), the leading developer, manufacturer and marketer of proprietary clinical grade cell and tissue hypothermic storage and cryopreservation freeze media (“BioLife” or the “Company”), today announced that its customer Kite Pharma, Inc., a wholly-owned subsidiary of Gilead Sciences, has received US FDA approval for Yescarta, the first CAR T-cell therapy for treatment of adult patients with relapsed or refractory large B-Cell lymphoma after two or more lines of systemic therapy. [Read more…]

FDA approval brings first gene therapy to the United States

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CAR T-cell therapy approved to treat certain children and young adults with B-cell acute lymphoblastic leukemia

U.S. FDA, August 30, 2017 – The U.S. Food and Drug Administration issued a historic action today making the first gene therapy available in the United States, ushering in a new approach to the treatment of cancer and other serious and life-threatening diseases. [Read more…]

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