ARTICLES BY ED MISETA

• Stem Cell Therapy Trial Offers Hope To Osteoarthritis Patients

  Cynata Therapeutics is producing products called mesenchymal stem cells (MSCs), which have huge therapeutic potential for numerous unmet medical needs. The company’s Cymerus technology addresses many of the challenges and complexities of manufacturing stem cells at commercial scale.

• Former Novartis CEO: Cell And Gene Therapies Will Drive Innovation

  Joe Jimenez joined Novartis as division head of Consumer Health. A few months later, he was asked to run the pharmaceutical division. Shortly after his promotion to CEO, Novartis was the first company to pursue a CAR T therapy. Today, Jimenez marvels at the innovation taking place in the industry, and believes cell and gene therapies will drive future development. 

• Cell & Gene Therapies Require the Right Tracking Solution

  In cell therapy trials, the ability to track a patient’s cells is essential. When you have over 100 patients, and the cells will require multiple shipments, using a spreadsheet is not possible. “For the safety of our patient, accurate tracking is a must,” says Gerald Garrett, VP of clinical operations for MarkerTherapeutics. “We need to know exactly where the product is at any step of the way.”

• NeuBase Prepares To Face The Challenges Of A Rare Disease Trial

  NeuBase is attempting to create a treatment for two rare diseases: Huntington’s disease and myotonic dystrophy. The company will face several challenges along the way, with the first being patient recruitment. Will young patients even want to know if they have the disease?

• GenSight Uses Gene Therapies To Target Neurodegenerative Diseases

  GenSight Biologics believes it is on the cutting edge of ophthalmology, gene therapy, and drug development. The company is focused on finding treatments for patients with neurodegenerative diseases and is developing products that are a combination of gene therapy, its mitochondrial targeting sequence technology, and optogenetics.

• The Cell Therapy Discovery About To Disrupt Clinical Trials

  While pursuing hid PhD in chemical engineering at MIT, Armon Sharei developed an interest in novel cell therapies. That interest led him to a discovery that today is the foundation of SQZ Biotech, a cell-therapy company where he serves as CEO. Sharei discovered a new method of inserting materials into cells more effectively than anything that currently existed.

• A Patient’s Journey: Human Interactions Matter In Immunotherapy Trials

  Mary Elizabeth Williams is a journalist and an author. She is also a patient who has with metastatic melanoma who participated in a Phase 1 immunotherapy clinical trial. There is much that pharma can learn from her in terms of how companies approach clinical trials and drug development.

• Manufacturing Facility Will Meet Needs Of Cell & Gene Trials

  Abeona Therapeutics is developing investigational gene and cell therapies for people living with serious and rare diseases. Last year, Abeona held a ribbon-cutting ceremony to celebrate the groundbreaking of a manufacturing facility that is one year later producing gene therapy material for their clinical and pre-clinical programs. As is the norm with gene therapy, Abeona uses a virus as vehicles to deliver functional copies of defective genes.

• Novartis Turns To Digital Technologies For Clinical Trials

  Jake LaPorte, head of digital development for Novartis, talks about how AI, IoT, and machine learning are all being leveraged for better clinical trials.

• A Patient’s Journey To Join A CAR-T Cell Therapy Trial

  Nicole Gularte is currently battling cancer for the 8^th time. The last time she had to do so, it seemed a CAR-T cell therapy trial might be the solution. This article is about her long battle with cancer, the struggle to get into a CAR-T cell trial, and the ups and downs she has faced along the way. Her story is an inspiration to others.

• Science Driving Gene Therapy Development At bluebird bio

  For patients with sickle cell anemia, just getting through the day can be a struggle. Symptoms of the disease include vision problems, swelling of the hands and feet, and periodic episodes of pain that can last from a few hours to a few weeks. In this article Nick Leschly, CEO at bluebird bio, discusses the progress being made with its investigational gene therapy for sickle cell disease.

• Is Genetic Testing Creating a Moral Dilemma For Clinical Researchers?

  Clinical trials are complex, but adding genomics to the equation has the potential to make them even more complicated. However, much of the genetic testing that is now being performed in the clinical space is done before a trial begins. That means companies need to do some thinking before adding a genetic test to the protocol.

• Ensure A Smooth Start-Up In Gene Therapy Trials

  Preparations for a large Phase 3 clinical trial will always present challenges. In the area of gene therapies, those challenges can be magnified. Gene therapy trials can add more time and cost to a trial, and will also require specific expertise not needed in small molecule and biologics trials.

• Cell Therapy Trial Creates Skin Cells For Rare Disease Patients

  Abeona Therapeutics recently conducted a Phase 1/2 clinical trial for individuals with Recessive Dystrophic Epidermolysis Bullosa (RDEB). RDEB is an often fatal connective tissue disorder marked by skin blistering and caused by a lack of a collagen protein responsible for anchoring the dermal and epidermal layers together.

• Cell Therapy Trials Present Unique Challenges For Pharma

  Maria Fardis, CEO of Iovance Biotherapeutics, has spent 18 years working in the pharma industry. Although most of her experience has been in small molecules and proteins, she now works in the cell therapy space and notes these treatments are a new and exciting space with meaningful differences in how clinical trials are conducted.