Biotech Strategy Blog

Commentary on Science, Innovation & New Products with a focus on Oncology, Hematology & Cancer Immunotherapy

Posts from the ‘CAR-T’ category

Now that at two CAR T cell therapies have been approved by the FDA in two indications, what does the future hold for new developments in both hematologic malignancies and solid tumours?

It was astonishing to explore the poster halls at AACR last month and see just how many new players and targets are emerging left, right and centre.

Last week we highlighted an up and coming new player on the scene, Mustang Bio, but what about the original pioneers in this niche and what are they up to these days?

To answer this question, we tracked down Dr Renier Brentjens at Memorial Sloan Kettering while in Chicago to learn more about his latest work and where he sees the future of CAR T cell therapy heading. It makes for a very interesting, and at times, surprising read…

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At the recent AACR meeting in Chicago one thing that was a surprise was how many new players seem to be emrging in the CAR-T cell space, not to mention the plethora of targets being evaluated preclinically in both hematologic malignancies and solid tumours.

The CAR-T cell niche is becoming very competitive and gritty

If we thought the market was becoming competitive before with less than a dozen players, imagine how crowded it will get once many of the unknowns start to make their mark?

This situation also presents many challenges and opportunities for the new entrants, not just in terms of merely identifying new targets and preclinical research, but also in the need for quality control and manufacturing expertise plus clinical development.

We should also remember that immunotherapy is designed not to target the tumour per se but unleashes the immune system on the tumour. This means that lessons from one approach (e.g. checkpoint therapy) can be applied to another (e.g. CAR-T cell therapy) and vice versa.

Yesterday, we discussed CD123 from the perspective of a bispecific company, what about approaching the target with a CAR-T cell therapy? What other alternative targets are out that that may be useful to investigate in the clinic?

We decided to explore these issues through the lens of one of the up and coming players in the CAR-T cell niche and find out more about what they are doing, how they see things evolving in this dynamic environment and what their path to market strategy is…

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Salt Lake City, Utah – The CAR T cell therapy niche has certainly provided plenty of controversy, highlights and lowlights over the last decade or so, although much of the mainstream attention has really only surfaced in the last couple of years.

Gone skiing?

For those interested in this space, there is a short synopsis of the BMT Tandem 2018 plenary session that took place this weekend and simultaneously published in the ASBMT journal (See: Perales et al., 2018):

“Building a Safer and Faster CAR: Seatbelts, Airbags, and CRISPR” 

While no one doubts that we have a need for safer CARs to reduce or ameliorate severe toxicities (the debate here is what are the best ways to achieve that in the clinic), it remains unclear whether a fast or slow approach is the optimal way forward in terms of efficacy.

In this post, we take a look at new clinical and scientific findings that may pave the way forward for the future in the CAR T cell space through the lens of several different academic institutions…

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Salt Lake City – at the 2018 BMT Tandem meeting (Twitter: #BMTTandem18) the combined annual meeting of the American Society for Blood and Marrow Transplantation (ASBMT) and Center for International Blood & Marrow Transplant Research (CIBMTR), one of the presentations of note today was a 7am breakfast symposium entitled:

Realizing the Promise of CAR T cell Therapy for Leukemia and Lymphoma: Implications for Long-term Care in the Era of Stem Cell Transplantation.”

Cancer cells in culture Source: Dr Cecil Fox, National Cancer Institute

This educational session supported by grants from Kite/Gilead and Novartis, featured two BMT transplant experts with hands-on experience of CAR T cell trials: Dr Stephan Grupp (@GruppSteve) from The Children’s Hospital of Philadelphia and Dr Krishna Komanduri (@drkomanduri) from the University of Miami Sylvester Comprehensive Cancer Center.

We’ve previously interviewed both Dr Grupp and Dr Komanduri on BSB, so were keen to hear how leading transplanters view the CAR T landscape now that two therapies have been approved by the FDA, and how they think this approach will integrate with transplants, and which patients will benefit most from this therapy.

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Bellicum have just announced that the FDA placed a clinical hold on BPX–501 clinical trials in the United States following three cases of encephalopathy “deemed as possibly related to” treatment with their new product in development, BPX–501.

The FDA clinical hold does not affect the ongoing BP–004 registration trial in Europe.

Here we take a look at some of the issues underlying the unfortunate news.

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Dr Michael Gilman, Obsidian

To get started in the New Year, we decided to roll out a new mini series on small cap and private biotechs, as seen through the lens of their dynamic CEOs.

This will run throughout January, with plenty of different products and perspectives to consider.

The honour for the very first CEO to be in the hot seat in 2018 went to Dr Nancy Simonian of Syros earlier this week during JPM18, who talks about their small molecule program and why they are excited about 2018.

When we first talked to Syros, they were a privately held start-up, now they’re listed on Nasdaq.

We now move on to the second one in the series, with a look at a new start-up called Obsidian Therapeutics in an interview with Dr Michael Gilman (right).

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For the last couple of years at every annual meeting of the American Society of Hematology (ASH) conference, I have posted an extensive Preview of the CAR T cell therapy landscape and looked at which abstracts piqued my interest.

The roaring 30s CAR

This year the review is the most extensive to date, with more companies, more research groups, more tumour types and way more preclinical research coming through. It’s like a kaleidoscope of ideas cascading through R&D.

The other thing to take note is how fast the field is moving – it’s warp speed now and so much comes through the literature every month on top of that.

So here we go – hold onto your hats as there is a LOT to contemplate this year!

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Dr Stephan Grupp at SITC17

National Harbor, MD – SITC 2017 wouldn’t be the same without an in-interview with Dr Stephan Grupp (CHOP) on the issues surrounding the Juno ROCKET trial and CAR T cell therapy safety.

This data has been eagerly awaited and anticipated by the field since the news of a cluster of lethal cerebral oedemas hit Juno in the summer 2016, seemingly out of the blue.

Yesterday, we reviewed the salient points from Dr Mark Gilbert (CMO) presentation highlighting their findings from the in-depth analyses performed to date.

In the second part we turn to a CAR T cell therapist with experience in treating children and young adults for his perspectives and candid reactions to the information presented.

We also talk about where the field is headed and some of the new developments we can look forward to hearing more about in the near to medium term future.

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Paris – amazingly it’s now 3 years since we interviewed Cellectis (NASDAQ: $CLLS) CEO André Choulika and CSO Philippe Duchateau (See post: Can Cellectis revolutionise CAR T cell therapy):

Cellectis CSO CEO

Cellectis Senior Management – Drs Duchateau and Choulika

Since then, we’ve followed the company over time, including an interview with one of their leading scientists, Dr Julianne Smith at ASH 2014, followed by the initial results of their first allogeneic CAR T cell therapy UCART19 presented at #ASH15 by Professor Qasim.

It’s hard to believe 3 years have gone by so quickly! As regular readers know what we often do on BSB is follow stories longitudinally, so while in Paris for an Immuno-Oncology Summit we thought it a rather timely opportunity to revisit Cellectis and take stock of where they’re at and ask what the future may hold for them?

With the recent news that Gilead have acquired Kite Pharma, there’s going to be a lot of interest in what companies such as Cellectis are doing to bring allogeneic “off the shelf” CAR T cell therapy to market.

This is the penultimate post in our summer mini-series on gene editing and allogeneic CAR T cell therapy and features a candid interview with Dr Philippe Duchateau, Chief Scientific Officer, at Paris based Cellectis.

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When we think of pioneers in the CAR T cell therapy space, one person who comes to mind is Waseem Qasim, Professor of Cell and Gene Therapy at the Institute of Child Health at University College London, and a Consultant Immunologist and Pediatrician at Great Ormond Street Hospital (GOSH).

Institute of Child Health

As readers may recall back in 2015, he gave the first allogeneic CAR T cell therapy under compassionate use to an infant with ALL, and in the process undoubtedly saved her life.

The subsequent case report published in Blood was the talk of 2015 annual meeting of the American Society of Hematology (ASH) in Orlando.

The poster focused on the first child that Prof Qasim treated and attracted a phenomenal amount of attention:

Prof Qasim UCART19 #ASH15 Poster

Where are we now with allogeneic CAR T cell therapy?

It’s been 18 months since we spoke to Prof Qasim, so while in London over the summer BSB caught up with him in his office at the Institute of Child Health.

This interview is the first in our latest 3-part mini-series on allogeneic CAR T cell therapy, which runs throughout this week. Here’s a teaser clip:

Kite’s first autologous product, Axi-Cel (in aggressive lymphomas), heads for regulatory approval in the US (PDUFA date November 29th), offering Gilead a hematology launch product with a high unmet need and, presumably, a relatively high price tag to match. Inevitably, some critical attention will subsequently be focused on the pipeline and whether they will move towards allogeneic CAR-T cell therapy (reduces cost of goods and increases profit margin) as well as how the TCR platform in solid tumours will fare.

It’s certainly a timely point to consider allogeneic CAR T cell therapies again given that things are rapidly heating up in the cell therapy niche following the Gilead announcement yesterday that they are acquiring Kite Pharma for $11.9 Billion.

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