Cell and Gene Therapies
Gene therapy and Cell therapy are overlapping fields of biomedical research with similar therapeutic goals to manipulate a patient's cells for the treatment of an inherited or acquired disease.
The Advocate of Rare Disease Advocates: Remembering Henri Termeer
We were saddened to come to work Monday morning to discover that innovator, industry giant and Genzyme founder Henri Termeer had died. We had the pleasure of working with Termeer over the years as...
Tissue regeneration: A vital area within regenerative medicine
Guest post by Patricia Reilly, M.S., Head of Intelligence Alliances and Unification, Pharma Intelligence and Nancy Dvorin, Executive Editor, In Vivo Cell and gene therapies often get the lion’s...
Bruce Levine is a man you can thank for your future cell therapy
To call Bruce Levine, PhD, an innovator in the cell and gene therapy space is a gross understatement. He is known for his groundbreaking work in the development and use of synthetic immune cells...
BIO-Europe Spring® 2016: CEVEC CEO talks US deals to expand business
Frank Ubags, CEO of CEVEC—a company offering industrial scale production of viral vectors for gene therapy firms—speaks with Scrip's Lucie Ellis about the types of deals the platform technology compan
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BIO-Europe Spring® 2017: Partnerships are key for rare disease drug discovery and development
What kinds of partnerships are key to getting rare disease research out of the lab and to patients as therapies and cures? Wendy White, Board Chairman of Global Genes, discusses her work on behalf of
Rare diseases, rare commitment
Patients, their families and patient foundations are invaluable in helping drug developers understand the day-to-day realities of approx. 7,000 rare diseases (of which only 400 have therapies)....
Rare Disease Foundation CEO cites collaboration, data sharing as keys to progress
An interview with Monica Weldon, President of Bridge the Gap – SYNGAP Education and Research Foundation Houston-based Monica Weldon is a mother, wife, former teacher and a rare disease advocate...
BIO-Europe Spring® 2017: Commercializing cell and gene therapies
New business models for clinical development and manufacturing require strategic partnerships among researchers, manufacturers and payers to fully realize the commercial opportunities.
Q1 2017 – Advanced Therapies Make a Strong Showing Early in the Year
Guest post by Patricia Reilly, M.S., Head of Intelligence Alliances and Unification, Pharma Intelligence and Nancy Dvorin, Executive Editor, In Vivo Advanced therapy companies started 2017 with a...
Can pharma afford to cure patients instead of treating them?
Who wants a one-shot therapy that delivers a durable response in patients? What is the price for a cure that payers are willing to pay? Why is pharma turning its attention away from this...
POLs: Patient opinion leaders emerge as key to success in orphan drug designation
Orphan drugs are wicked hot. Wall Street loves them. And patients are a powerful force that can make or break a decision to grant that coveted designation for your drug. But don't count on having...
Realities of Rare Disease and the Role of Patient Investors
Guest post by Nicole Boice, Founder & CEO, Global Genes – Allies in Rare Disease Rare Disease represents the largest disease community on the planet! With over 7,000 identified diseases,...
2016 – A Year in Review for Advanced Therapies
Guest post by Patricia Reilly, M.S., Head of Intelligence Alliances and Unification, Pharma Intelligence | Informa The past year saw intensifying dialogue on how to assess the value of and...
World RARE Disease Day shines a light on rare disease community
Tomorrow is the 10th annual World RARE Disease Day. Various activities are taking place all over the United States as well as countless locations around the world. World RARE Disease Day is an...
Unmet needs, uncommon commitment: The shift toward investment in rare diseases
Join biotech leaders, emerging innovators, investors and patient advocacy group reps for a candid, interactive and enlightening discussion about rare disease drug discovery and development. Read...
The state of cell and gene therapy: Reimbursement and manufacturing dominate
The number of cell therapies in clinical trials has grown 21 percent since 2015, with more than 759 trials underway globally. With that growth it is time for the industry to get serious about...
The renaissance of gene therapy
Gene therapy and gene editing are at the beginning of a renaissance, according to speakers at Biotech Showcase™ 2017. While sizeable challenges exist, they are being overcome, leaving the field...
BIO-Europe® 2016: Immunotherapies: Partnership models driving cures
While we have seen new immunotherapies enter the market and there are many exciting products in development; we have also seen recent setbacks. The field is risky and partnerships among biotechs, phar
BioPharm America™ 2016: Novel approach to CAR T cell therapy at UPenn Center for Cellular Immunotherapies
Avery Posey, Instructor at the Center for Cellular Immunotherapies at the University of Pennsylvania is interviewed by Samuel Murphy, Managing Partner at BioPharma Capital, Inc., about his work in CAR
A look into Intellia’s strategy for “fixing the broken gene”
The field of genome editing is hot and growing. Despite legal battles over key patents on the CRISPR technology, the important work of genome editing goes on. Even the layperson can understand the...