This week's life sciences industry news is heavy on gene therapy with an important anniversary in the field, a pioneer being honored, a manufacturing partnership to support developers, and more.
Here's the roundup:
Children's Hospital of Philadelphia
Emily Whitehead, the first child in the world to receive CAR T-cell therapy, is celebrating her 10th year of being cancer free.
When she was 6 years old and under the care of Children's Hospital of Philadelphia's Dr. Stephan Grupp, she was the first patient to enroll in the clinical trial for CAR T-cell therapy in pediatric patients with acute lymphoblastic leukemia, making her the first pediatric patient ever to receive the treatment.
CAR T-cell therapy is designed to improve the ability of a patient’s own immune system to fight cancer by reengineering their T cells in the lab, then putting those cells back in the patient to attack proteins found on the surface of cancer cells.
Grupp, a pioneer in the field of cellular immunotherapy, worked with researchers Drs. Carl June, Bruce Levine and David Porter at Penn Medicine’s Abramson Cancer Center to establish a protocol to infuse Whitehead with their experimental CAR T-cell therapy product targeted against leukemia.
Whitehead's family brought her to CHOP after she relapsed and was no longer eligible for a bone marrow transplant — and her local medical team had told her family there was nothing left to do.
The CAR T-cell therapy treatment worked and she remains cancer free.
“What we learned from Emily has defined the entire field of CAR T-cell therapy,” said Grupp, director of the Susan S. and Stephen P. Kelly Center for Cancer Immunotherapy at CHOP. “Ten years ago, we had no idea what to expect. Would the treatment work? Would it last? In the end, her outcomes far exceeded our most optimistic expectations – not only did the treatment work for completely uncontrolled disease, but her engineered T cells endured and prevented relapse for what has now been 10 years. We have since treated more than 440 patients at CHOP with this therapy, and thousands of pediatric patients around the world have received it as well. It has truly been a revolution in pediatric cancer care, and it started with Emily.”
More CHOP
The West Philadelphia pediatric medical center has awarded its Gold Medal to Dr. Katherine High for her pioneering work in the field of gene therapy.
CHOP has only given this award 12 times in its 167-year history.
High’s groundbreaking discoveries at CHOP led to a gene therapy treatment – Spark Therapeutics' Luxturna product – for a rare form of inherited blindness and advanced to late-stage testing the development of a gene therapy for hemophilia.
In 2013, High co-founded Spark, a CHOP spinoff company, with Spark's former CEO Jeff Marrazzo. Last year, she took a new job as president of therapeutics at North Carolina-based Asklepios BioPharmaceutical Inc.
Avantor
The Radnor-based chemicals and materials company is teaming up with Cytovance Biologics Inc., a biologics contract development and manufacturing organization, in a partnership to make and supply plasmid DNA for biopharma customers.
Plasmid DNA is an important process element used in viral vector-based gene therapy and mRNA vaccines and therapeutics.
This partnership will allow Avantor (NYSE: AVTR) to provide global biopharma customers and contract manufacturing organizations access to plasmid DNA and other materials used in research, clinical testing and manufacturing scale-up and commercialization.
Terms of the deal were not disclosed
Dr. Ger Brophy, executive vice president for biopharma production at Avantor, said demand for therapeutic biologics in the gene therapy space is increasing rapidly, and will require increased manufacturing capacity and expertise. Brophy said the collaboration shows the company's commitment to cell and gene therapy biomanufacturers, and will support Avantor's role in "unlocking the potential these therapies hold for patients around the globe."
Quick Hits
The Food and Drug Administration approved Olumiant, a drug developed by Eli Lilly and Co. (NYSE: LLY) and Wilmington-based Incyte (NASDAQ: INCY) to treat Covid-19 in hospitalized adults requiring supplemental oxygen, mechanical ventilation or advanced oxygenation. … The University of Delaware and Waters Corp. (NYSE: WAT) of Milford, Massachusetts, opened Immerse Delaware, an innovation and research laboratory on the university’s Science, Technology and Advanced Research campus. The project is part of a multiyear research partnership the two organizations formed in October 2021 to foster collaborations with biopharma scientists both in industry and academia to advance industrial processes for making and characterizing biotherapeutics. Waters is a global analytical instruments and software company that has 14 manufacturing facilities worldwide. … Radnor-based NRx Pharmaceuticals (NASDAQ: NRXP) enrolled its first patient in one of two psychiatry studies being initiated this year. The first trial is studying the effectiveness of its new drug candidate NRX-101 in patients with bipolar depression and sub-acute suicidality. The company plans to start a second trial of the experimental therapy in patients with bipolar depression and acute suicidal ideation and behavior later this year. … Lannett Co. Inc. (NYSE: LCI), a Trevose-based generic drug company, entered into an agreement with Indiana-based Areva Pharmaceuticals to be the exclusive U.S. distributor of fludarabine phosphate single-dose vials, a treatment for leukemia. The product is a generic version of Bayer AG's branded Fludara product. Financial terms of the deal were not disclosed. … Lava Therapeutics (NASDAQ: LVTX), which has operations in the Netherlands and Philadelphia, has received FDA approval to begin human testing of LAVA-051, the company's experimental treatment for relapsed or refractory chronic lymphocytic leukemia, multiple myeloma and acute myeloid leukemia.
