The ‘transformative power’ of gene editing, with David Sourdive, Cellectis

‘Gene editing has a very transformative power in adaptive T cell therapy. In gene editing, we can make T cells do much more than they would normally do without being engineered.’ In this far-ranging interview, David Sourdive, Executive Vice President of Corporate Development at Cellectis, outlines the exciting updates from Cellectis’ current CAR-T therapy projects, from manufacturing, to clinical trials. He details his hope for the future and highlights the challenges that working with T cells can bring.

The possibilities of using gene editing to leverage the power of T cells into ‘better killers’ to ‘overcome the defence mechanisms of tumour cells’ are clear for Sourdive. Whilst acknowledging the current successful use of autologous treatments, he believes the industry is moving towards the possibility of ‘real pharmaceutical off the shelf T cell products’. There is an undeniable enthusiasm from Sourdive about gene editing to ‘maximise the power of T cells’, which can develop treatments that will meet the standard of care that chemotherapies and antibody therapies have.

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Biosimilars Are Here. Now What?

More and more biosimilars enter the market raising questions around pricing, regulations, but also how to convince physicians to start using biosimilars, and how to switch the patients.

This is an excerpt from a panel discussion with Jim Roach of Momenta Pharmaceuticals, Inc., Irena Royzman of Patterson Belknap Webb & Tyler LLP, Sophie Opdyke of  Pfizer, Chrys Kokino of Mylan, and was moderated by John Farah of Red Team Associates. This panel of experts shed light on the following topics:

  1. The benefits and risks associated with bringing biosimilars into the U.S. market.
  2. What do we need to do to accelerate the timeframe of how we accept biosimilars in the U.S.?
  3. Because of the high costs, how do we increase support to get these products into the country?

View the complete discussion.

Genetic Engineering of Red Blood Cells for Therapeutic Function

Robert J. Deans Ph.D, Chief Scientific Officer at Rubius Therapeutics, a Cambridge Massachusetts based biotechnology company, started the Emerging Research: Delivery Strategies and Genome Engineering Technologies session by introducing Rubius Therapeutics’ work developing red blood cells (RBC) as a therapeutic agent. Rubius Therapeutics is a Flagship VentureLabs sponsored company that arose from research originating out of Harvey Lodish’s and Hidde Ploegh’s laboratories at MIT. Rubius Therapeutics’ goal is to develop RBCs as a therapeutic delivery vehicle for the treatment of chronic and acute diseases. According to Robert J. Deans, RBCs show great potential as a delivery vehicle as they have have a long persistence time, show a predictable bio-distribution, have a natural immune evasion capability, and confer no risk of oncogenicity. He also spoke at length on the company’s overall process of growing cells derived from Human Stem Cells in vitro and then engineering them to express desired genes so that the product, produced and retained in the RBC, is then delivered by the RBC to the target cells. He finished his talk by presenting research results from a study that used the RBC delivery system to treat phenylketonuria, a disease of the metabolism.

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The New Age of CRISPR

The federal biosafety and ethics panel has unanimously approved the first study in patients of the genome-editing technology CRISPR-Cas9, in an experiment that would use CRISPR to create genetically altered immune cells to attack three kinds of cancer.

It had been widely expected that the first human use of CRISPR would be a 2017 clinical trial by Editas Medicine, which announced last year that it plans to use CRISPR to try to treat a rare form of blindness called Leber congenital amaurosis. Only a few hundred people in the US have that disease. The possibility that a study siccing CRISPR on cancer will happen first suggests that the revolutionary genome-editing technology might be used against common diseases sooner than once thought.

The experiment, proposed by scientists at the University of Pennsylvania, still needs the approval of the medical centers where it would be conducted, as well as from the Food and Drug Administration, which oversees the use of experimental treatments in people. If the study gets those OKs, it would enroll patients with multiple myeloma, melanoma, and sarcoma, and be funded by the Parker Institute for Cancer Immunotherapy, which was launched this year by tech mogul Sean Parker.

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Bringing Orphan Drugs and Gene Therapies to Market: Maximising ROI through an Effective Communications Plan

Businessman with business plan concept analysis strategy questions

Howard Sinclair, Strategic Director – Rare Diseases and Gene Therapy, The Prime Medical Group, UK, and Andrew Jobson, PhD, Senior Editorial Manager – Rare Diseases and Gene Therapy, The Prime Medical Group, UK, are talking at Cell Therapy Manufacturing & Gene Therapy Congress on Wednesday 3rd February in Brussels. Here they explain what they’ll be talking on:

“There are a multitude of urgent and critical areas to address in further developing your orphan drug or gene therapy to achieve the next milestone. They consume all your team’s energy, resources and time, and there never seem to be enough hours in the day. Against this reality, is creating a communications plan really that important? – Yes! Won’t the external communications just happen naturally anyway, as the relevant data become available? From decades of experience supporting everything from mega-blockbuster first-in-class agents, to very specialised medicines for the most obscure ultra-rare diseases, we would respectfully and emphatically answer: No.

Unique challenges exist for rare diseases, orphan drugs and gene therapies that require tailored solutions, distinct from standard approaches. Creating a robust, evidence-based, authentic and compelling scientific narrative is the starting point – and you will need a sound plan to effectively share this with potential product users, key influencers and the broader healthcare community. This could include clinical trial investigators, practicing specialty clinicians, primary care providers, nurses and payers.

An effective strategic communications plan is critically important, enabling you to optimally engage with your target audiences at the right time (which will likely vary over the course of the development of your asset) to achieve your immediate and long-term goals, whether it’s accelerating clinical trial accrual or ensuring optimal awareness and adoption into clinical practice upon market authorisation. Early development and initiation of a tailored, proactive and cohesive communications plan is arguably even more critical for orphan drugs and gene therapies to ensure your limited resources are most effectively channelled.

Topics to be covered will include:

  • Adding value to your product/clinical studies programme with a tailored medical communications plan.
  • Addressing the specific challenges for a medical communications plan in orphan drugs, rare diseases and gene therapy.
  • Driving the communications strategy through a focus on the unmet needs of both the patient and the healthcare professionals.
  • Defining the key elements of an optimal communications plan.
  • Making the plan happen – driving the initiation, management and delivery.

Creating a robust and tailored strategic communications plan is critical to success – we will explore the why, what and how.”

See the full agenda for Cell Therapy Manufacturing & Gene Therapy Congress and buy tickets at  

Revolutionary Algorithm Mogrify Set to Transform the Field of Regenerative Medicine

Credit: Nature Genetics & Rackman et al.

This week a paper published by a team of international researchers at the University of Bristol has sent excitement rippling through the cell therapy community. The team comprised of collaborators from Bristol, Australia, Singapore and Japan published the breakthrough last Monday (18/01) in Nature Genetics. They presented the creation of a predictive system (Mogrify) that can forecast how to create any human cell type from another cell type directly, bypassing the need for exhaustive trial and error.

The team led by Julian Gough have so far applied Mogrify to 173 human cell types and 134 tissues, outlining an index of cellular reprogramming.  Speaking about the breakthrough Gough, professor of bioinformatics, revealed to the University of Bristol that “the barrier to progress in the field is the very limited types of cells scientists are able to produce. Our system, Mogrify, is a bioinformatics resource that will allow experimental biologists to bypass the need to create stem cells”.

Secondly, when listing further achievements from the research, conducted in collaboration with Professor Jose Polo at Monash University in Australia, Gough confirmed that Mogrify had validated two new transdifferentiations. The algorithm succeeded first time in validating both of the new transdifferentiations, and it is this speed in achieving results that lends clear indication to the claim of Mogrify being revolutionary. Professor Gough added that he hoped “Mogrify will enable the creation of a great number of human cell types in the lab”.

This is a huge result for the regenerative medicine field and will no doubt speed up advances in life-changing medicines. Particularly, the ability to produce a number of types of human cells will directly lead to new tissue therapies, and a much improved understanding of cell production at a molecular level. One hope going forward is the potential to grow whole organs from someone’s own cells.

For five years Gough collaborated with Dr Owen Rackham to create a computational algorithm to predict the cellular factors for cell conversions. This was achieved largely in thanks to data collected as a part of the FANTOM international consortium, of which Gough is a member.

To highlight the size of the achievement, it must be remembered that scientists have only been able to discover conversions of human cells a handful of times since Japanese researcher Shinya Yamanaka created the first human artificial pluripotent stem cells in 2007.

The algorithm has been released and made available online for others to use, so that the field may advance at a much faster pace.

The paper ‘Mogrify: An Atlas for Direct Reprogramming Between Human Cell Types’ by Rackham et al in Nature Genetics.