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.
Jake LaPorte, head of digital development for Novartis, talks about how AI, IoT, and machine learning are all being leveraged for better clinical trials.
Nicole Gularte is currently battling cancer for the 8th 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.
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.
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.
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.
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.
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.
The watchdog group Center for Responsible Science (CRS), along with clinical trial participants and the father of a deceased trial participant, has filed a lawsuit against the FDA for denial of CRS’ citizen petition, which was originally submitted in June 2014 and has since had several amendments.
Getting adult patients to take part in a clinical trial is difficult. When developing a treatment for a childhood disease, that recruitment challenge becomes even more difficult. The combination of patient age and the experimental nature of a treatment can cause concern amongst both parents and the FDA. Caladrius Biosciences is a company dealing with that challenge. The company is developing a cell therapy technology based on a patient’s own T-cells.
Patient recruitment has always been a challenge for companies conducting clinical trials. But when you are developing treatments in the rare disease space, finding patients to take part in your trials becomes an even greater hurdle to overcome.
With the number of biologics now in development and soon to be making their way into clinical trials, the preservation of cells, tissues, and organs are suddenly increasing in importance. Companies handling the manufacturing, storage, and transportation of these materials need to be focused on improving the yield and extending the shelf life of these time and temperature sensitive biologics.
Abeona Therapeutics is named after the Roman goddess who was the protector of children as they start out on their journey. Since launching as a private company in 2013, Abeona has partnered with a dozen international foundations to fund its development efforts. Currently it is seeking a treatment for children with Sanfilippo Syndrome, and engaging caregivers to determine how to best meet the needs of patients taking part in trials.
Genelux is an oncolytic virus company that was formed in 2001 and has been conducting clinical trials since 2009. The company completed four Phase 1 studies which have enabled it to design what CEO Thomas Zindrick describes as an advanced clinical program.
In May 2014, I interviewed Mithra Bindhu of India-based Asiatic Clinical Research to get an update on performing clinical trials in that country. The DCGI (Drug Controller General of India) had recently attempted to overhaul the entire system, which took a long time, complicated the process of conducting trials in India, and caused some sponsors to question the value of doing business there. Has the situation changed?