‘If one or two CAR-T therapies get to market it will provide impetus and momentum behind the industry’ – Dr Akshay Peer, Vice President of Sales and Account Management at TrakCel discusses the revolutionary steps that cell and gene therapies are making through their increased commercialisation.
In a field that is constantly seeing developments, Dr Peer outlines CRISPR and CAR-T therapies as forms of gene editing that can dramatically change the face of gene therapies and their use within modern medicine. He believes that as the commercialisation of these treatments increases, the regularity of their use will as well.
This is due to the surge in ‘positive public opinion’ that will arise from the successful implementation of the therapies. Dr Peer identifies the positivity that already exists around the new therapies, with ‘everyone looking at when these therapies will come to market and how much they will cost’. Although not going into detail about the financial outcome, he outlines his hope that when the public can see the lives of adults and children enhanced by using these therapies, any uncertain opinions will change to ones of optimism and confidence.
Peer acknowledges the lack of information that can exist around the new therapies due to an unfamiliarity in their usage; ‘People can sometimes get ahead of themselves and not understand completely what we’re trying to do here in this industry’. However, he is sure that this will change once there is evidence of patients who are successfully treated.
There is clearly excitement developing around the use of gene therapies, outlined by Dr Peer.
Watch the full interview, filmed at Cell Therapy Manufacturing & Gene Therapy Congress, with Dr Peer above or here.
Recent clinical trials in the field of cell and gene therapy demonstrate remarkable therapeutic benefits with excellent safety. Despite demonstrated therapeutic effects, the commercialization of cell and gene therapies and their patient outreach remain scarce. Much of the research and development on cell and gene therapies is performed either on an academic level or by small and medium enterprises, largely excluding large pharmaceutical companies. Regulatory approval for cell and gene therapeutic products is performed on an individual product basis and is classified based on the degree of manipulation and intended end use. The primary deterrents to the lackluster commercialization of cell and gene therapies include the inherent complexities of the cells, issues with scalability for manufacturing and logistics and the complex regulatory requirements and time-consuming clinical trials. Cell and gene therapy products also have to navigate through the disparities in the regulatory requirements across regions. Furthermore, the complexity of product classification, extra requirements for combination of cell and gene therapies with a medical device, extensive paperwork surrounding the often ambiguous certification procedures and most importantly, the lack of harmonization of regulations across regions deter new investments and innovation in the field.
Limited understanding of the complex interactions of cell and gene therapeutics, absence of established standards and relatively scarce research data on the mechanism of action of these therapeutics make it difficult for stakeholders to navigate the complex and stringent regulatory requirements.
We elaborate the fundamental regulatory concerns associated with the development of cell and gene therapy products, and the need for international harmonization of regulatory requirement for approval of cell and gene therapies. The paper also addresses specific regulatory aspects in the EU and Japan as well as the roll-out of fast track mechanisms for market authorization in the EU and Japan. Finally, the paper addresses the urgent unmet need to provide regulatory certainty in the field of cell and gene therapeutics in the fast evolving global regulatory landscape.
The global market for cell – based therapies is expected to surpass the $20 billion USD mark by 2025, with an annual growth rate of 21%. The main targets for cell – based therapies are high impact disease areas with significant unmet needs, including cancer, heart disease, neurodegenerative diseases, musculoskeletal disorders and autoimmune diseases.
Examples of the landmarks attained are: 1) In order to accelerate a cure for diabetes, Harvard Stem Cell Institute, and Semma Therapeutics developed a process to generate nearly unlimited numbers of beta cells for the clinic. 2) Vital Therapies enrolled First Patient in Phase III Liver-Treatment Trial, for company’s ELAD System, an extracorporeal human allogeneic cellular liver therapy (already tested successfully in over 250 patients). 3) PCT (Caladrius Biosciences, Inc. , NJ, US) enter an agreement with Kiadis Pharma (Netherlands) on engineering and process development for a Phase III trial of Kiadis’ lead product, ATIR101™, for the treatment of blood cancers, in US and Canada. 4) A broad collaboration agreement was established between Memorial Sloan-Kettering Cancer Center (MSK) and Seres Therapeutics, Inc. to develop microbiome therapeutics for hematopoietic stem cell transplantation (HSCT) and immuno-oncology treatment in patients with dysbiotic microbiome. The FDA has granted SER-109 (Seres’ most advanced program) ‘orphan drug’ and ‘breakthrough’ therapy designations. 5) Vital Therapies bio-therapeutic company enrolled first patient in phase III liver-treatment trial. The study will exploit company’s ELAD System, an extracorporeal human allogeneic cellular liver therapy. 6) SAB Biotherapeutics with the US Navy has successfully developed a transchromosomic (Tc) bovine platform technology to produce Human Polyclonal Antibodies against Ebola Zaire Virus. Continue reading “Commercialisation of Cell and Gene Therapies [Whitepaper]”
As a lead in to the upcoming Cell & Gene Therapy Bioprocessing & Commercialization event this coming October, we are able to provide our readers with exclusive insights from some of the brightest minds in the industry. Today, Dr. Mehendra Rao, Vice President of Regenerative Medicine at Q Therapeutic and The New York Stem Cell Foundation, discusses the biggest trends, innovations, and challenges that are currently present but also on the horizon for the industry.
Here is what Dr. Rao had to say…
Continue reading “The Biggest Trends, Innovations, and Concerns in the Stem Cell Industry”
Taken from Christopher V. Gemmiti’s speech at our last Cell Therapy Manufacturing & Gene Therapy Congress, he spoke at length on the state of the industry, and how far we have come since the early 90’s and where we now find ourselves. Here’s what Christopher had to say.
Continue reading “State of the Industry: Innovation and the Hype Cycle”
DOWNLOAD THE GUIDE: How To Get Your Cell Therapy Research Funded
Getting funding is one of the most stressful and time-consuming parts of any researcher’s job. At last year’s Cell Therapy Bioprocessing & Commercialization conference, Brock Reeve, executive director of the Harvard Stem Cell Institute, gave the audience some advice on funding early stage science. He explained that because of uncertainty in financial markets, venture capitalists and other financially motivated investors have moved away from cell therapy, leaving a gap in funding. But this gap is being filled by venture philanthropy organizations who are moving things from the academic world into the commercial market.
This eBook offers a list of potential organizations who may be able to help fund your cell therapy research. It is by no means exhaustive, but hopefully it will make searching through the hundreds of pages of information out there a little bit easier, so you can get to the important stuff, like advancements in research, drug development, treatment, and potential cures.
DOWNLOAD THE GUIDE: How To Get Your Cell Therapy Research Funded
Join 1,700+ bioprocessing professionals at Biotech Week Boston on October 4 -7 2016 – find out more here.
Fabio D’Agostino, Biopharmaceutical Bioprocessing Technology Centre, Newcastle University, reported from Cell Therapy Manufacturing and Gene Therapy Congress in February:
Manufacturing biological medicinal products by the tonne might sound easy today but, as a recent Genentech infographic explained, the industry struggled to produce even grams of antibodies 30 years ago.
The dream of a second biotech revolution triggered by advancement in cell and gene therapies is driving big and the smaller pharma to find solutions to current manufacturing challenges that affect this new class of medicinal products.
Christopher Bravery (Director, Consulting on Advanced Biologicals Ltd., Member, ISCT EU Legal and Regulatory Affairs Committee, UK) opened the Cell Therapy Manufacturing and Gene Therapy Congress and introduced the first plenary talks from Johnathan Appleby (MDL and CSO, Rare Diseases Gene Therapy, GSK) ,Giovanni Milazzo (Head Regulatory Affairs Advanced Medicines, Chiesi Farmaceutici) and Diego Ardigo’ (Project Leader, Cell and Gene Therapy Products,Chiesi Farmaceutici). Very different companies but with a very similar challenge: turning data from research in academia and clinical environment into a MAA and therefore a commercially valuable cell medicinal product.
Continue reading “The State of the Industry: Cell Therapy Manufacturing and Gene Therapy Post-Conference Report”