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Top Ten Events in Advanced Therapies 2019

3 February 2020

From patent infringements, a flurry of M&AS, CRISPR  trials in humans; Phacilitate’s annual top ten list was revealed at Phacilitate leaders world. What would be on your top ten list of 2019?

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“We want to become the first, not the last therapeutic option.”

A remark that reverberated around the plenary hall at last week’s Phacilitate Leaders World in Miami. Bob Preti opened the conference with his take on where the advanced therapies industry finds itself today in comparison with some of the ‘big dreams’ from his 37 years in the industry. One thing that is clear is the expectations on progress have not been met. Bob also touched on the industry’s infrastructure, which is way behind the curve.

“This begs the question… what could we be doing better?”

This is a great question for a room full of industry leaders who all want to improve. Improve processes, improve science and, above all, improve the healthcare paradigm with life-changing and potentially curative therapeutics.

During the super plenary the previous evening, Anthony Davies introduced Dr Phillipe Menasche with a profound statement ‘by failing to study history, we are doomed to repeat it.’ I’m not sure that 2019 counts as history just yet, however, a feedback loop of learnings and understanding the bottlenecks and opportunities in front of us is a commendable exercise. Thank you to Susan Nichols and the advisory committee for shaping this list.

Let’s start with a recap of the number one spots from the past six years:

Top Ten Events in Advanced Therapies

The top ten events of 2019 are as follows…(you can watch the video if you prefer!)


One of the final events of the year was the closure of the high-profile patent dispute between Bristol-Myers Squibb’s Juno Therapeutics and Gilead’s Kite Pharma. The result was that Bristol-Myers won $752 million in this U.S. patent case after a jury in Los Angeles determined that Gilead’s Yescarta infringed on a patent licensed by BMS. This $752 million total is made up of a $585 million upfront fee, plus royalty on sales to date.

The lawsuit is based on a claim that Yescarta infringes on a patent that BMS licences from Memorial Sloan Kettering, which served to give BMS exclusive rights to the technology.

Top Ten Events in Advanced Therapies

Of course, there have been post-trial motions and statements on both sides and it’s doubtful that this is the end of the story. Gilead wholeheartedly disagrees with the outcome stating that “…we do not believe Sloan Kettering and Juno are entitled to any level of damages.” Whereas, BMS is looking for a punitive award equal to the value of this compensatory award.


2019 saw us take one step closer towards the promise of CRISPR technology. The first company-sponsored clinical trial of a gene therapy edited using CRISPR/Cas9 technology dosed its first patient.

In February 2019, CRISPR Therapeutics and Vertex infused the first patient in their phase I/II study of CTX001, used to treat transfusion-dependent beta-thalassemia.

This is certainly a positive sign of progress amongst the recent controversies faced by CRISPR technology. the ‘CRISPR patent war and the misuse of CRISPR by Chinese scientist He Jiankui, both of which were featured in last year’s the top ten events list (here’s a reminder).

Top Ten Events in Advanced Therapies


The flurry of announcements and commitments made by pharma and biotech to secure owned, internal manufacturing capacity gathered pace to become more of an avalanche throughout 2020! Reliable and strategic manufacturing capacity continues to be one fo the most significant bottlenecks for our industry and we’re seeing some big players spreading their risk and opting for internal capacity over or alongside CDMO partnerships. Here are two that Susan highlighted during her presentation:

Novartis took a two-pronged approach to increasing and protecting its cell and gene therapy manufacturing capacity. First, with the acquisition of CellforCure and their 3,600m2 facility in Les Ulis, France along with the 79 million Euro facility in Stein, Switzerland.

Pfizer invested a substantial $500 million to expand a gene-therapy manufacturing facility that it acquired when it bought Bamboo Therapeutics back in 2016. The facility, in Sandford, North Carolina, is Pfizer’s solution to improving yields, purity and product characterisation. This move also complements Pfizer’s recent acquisitions and partnerships that have given the company three clinical and ten preclinical gene-therapy programs.

Other organisations who have built or are building facilities include:

  • BlueBird
  • Precision
  • Kite
  • Audentes
  • Elevate
  • RegenxBio

On December 7th, Janssen presented some hugely promising data from their phase 1b CARTITUDE-1 study at the 61st ASH annual meeting in Orlando, Florida.

The company announced initial results from the study demonstrating the efficacy and safety of JNJ-68284528 (JNJ-4528), an investigational B cell maturation antigen (BCMA)-directed CAR-T therapy, which is being evaluated in the treatment of patients with relapsed or refractory multiple myeloma.

This phase 1b study involved a total of 29 patients treated with a median administered dose 0.73×106 CAR+ viable T cells/kg. Another important factor is that of this patient population, 86% were triple refractory. The results showed a staggering 27 (93%) showing progression-free tumours at six months, which is an encouraging sign for the durability of the treatment. Tumours were shown to shrink in 100% of patients and completely eliminated in 69% of patients.

Along with these excellent early results, there were, of course, some common adverse events, cytokine release syndrome being the most severe with one patient experiencing grade 3 CRS and one patient fatality from grade 5 CRS at day 99.

Overall, this was a strong dosing exercise for phase II, and based on the phase 1b results, a recommended phase II dose of 0.75×106 CAR+ viable T cells/kg was confirmed.  In addition, these data supported the recently announced U.S. Food and Drug Administration Breakthrough Therapy Designation for JNJ-4528.

Janssen has fully enrolled the phase II study with plans to report data by the end of 2020 so we’ll be watching this one with baited-breath and hope to see progress continuing.


In September 2019, Vertex Pharmaceuticals acquired Semma Therapeutics, a privately held biotechnology company pioneering the use of stem cell-derived human islets as a potentially curative treatment for type 1 diabetes, for $950 million in upfront cash for the outstanding Semma shares.

Semma’s value comes from two major scientific advances; the ability to produce large quantities of functional human pancreatic beta cells that restore insulin secretion and ameliorate hypoglycemia in animal models, plus a novel device that encapsulates and protects these cells from the immune system, enabling durable implantation without the need for ongoing immunosuppressive therapy.

Vertex already has a vast portfolio of Cystic Fibrosis therapies, with three approved therapies and, most notably, Trikafta, the triple combination therapy that is expected to be able to treat 90% of cystic fibrosis patients. With cystic fibrosis-related diabetes affecting 40-50% adult patients, it’s a sound, strategic acquisition for Vertex, as described by Vertex’s CEO, Jeffrey Lieden.

“This acquisition aligns perfectly with our strategy of investing in scientific innovation to create transformative medicines for people with serious diseases in speciality markets,” said Jeffrey Leiden, M.D., Ph.D., Chairman, President and Chief Executive Officer of Vertex. “We are excited to work with the talented scientists at Semma to build on their significant progress toward providing effective and potentially curative cell therapy options for people living with type 1 diabetes. We see a substantial opportunity to transform the treatment paradigm for type 1 diabetes, a speciality disease cared for by endocrinologists, both by advancing the development and manufacturing of the cells themselves, as well as through the highly innovative cell/device combination.”


December saw an end-of-year buying spree for Astellas, who announced two major acquisitions. They bought Audentes Therapeutics for $3 billion and Xyphos for up to $665 million, placing a big bet on gene therapies and bolstering their immuno-oncology pipeline, respectively.

These events seem to signify that Japanese pharma remain heavily invested in cell and gene therapy and that medium pharma may be using advanced therapies strategically to grow and expand.


It’s not just pharma and bio that are expanding their capacity with vigour. CDMOs are rapidly seizing the opportunity to create more, specialised capacity and gain more market share globally.

There were two billion-plus acquisitions this year from Thermo Fisher and Catalent. We also saw a triple-pronged approach from Fujifilm with a) a $890-million acquisition of Biogen’s large-scale biomanufacturing facility, b) the new $21 million FujiFilm Cellular Dynamic Centre in Madison, Wisconsin and c) a $91.9 million expansion of FujiFilm Diosynth’s site in North Carolina.

Here’s an overview of the landscape in 2020:

  • Catalent-Paragon $1.2 billion
  • Thermo Fisher-Brammer Bio, $1.7 billion
  • Hitachi-apceth, $86 million
  • Fujifilm-Biogen, $890 million
  • Cognate-Cobra, undisclosed

The wave of cell and gene therapy deals we’ve seen this year and prior has consisted mostly of partnerships and licensing deals rather than mergers or acquisitions. This could be due to the lack of long-term safety and efficacy data associated with the field today, but it also may be a reflection of the unique challenges in the development of advanced therapies.

Nevertheless, 2019 saw some significant licensing deals that are sure to harness to power of partnerships.

At the beginning of the year, Genentech and Adaptive Bio entered into a license agreement worth up to $2 billion, with $300m upfront plus milestones, to develop, manufacture and commercialise novel neoantigen directed T-cell therapies for the treatment of a broad range of cancers. The collaboration will combine Genentech’s global cancer immunotherapy research and development leadership with Adaptive’s proprietary T-cell receptor (TCR) discovery and immune profiling platform (TruTCR™) to accelerate a transformational new treatment paradigm of tailoring cellular therapy for each patient’s individual cancer.

The agreement also states that Genentech retains control over future commercialisation, and Adaptive is barred from developing T-cell receptor-based cell therapies in oncology on its own or with a third party.

Here are some other significant licensing deals from 2019:

  • Roche is paying $1.15 billion upfront for the ex-U.S. rights to Sarepta Therapeutics’ Duchenne muscular dystrophy (DMD) gene therapy
  • Vertex and CRISPR Therapeutics have expanded their collaboration and entered into an exclusive licensing agreement to discover and develop gene-editing therapies for the treatment of DMD and DM1. CRISPR will receive an upfront payment of $175 million, with potential for additional milestone and royalty payments


Susan summed this up perfectly in her presentation. “When I look at this, I think it signals that big pharma is optimistic towards M&A in the advanced therapies space and the value these therapies can create.”


Big pharma certainly seems to be clawing for a piece of the advanced therapies pie and putting infrastructure in place to develop and commercialise advanced therapies, But is big pharma set up for an advanced therapies business model, are they adaptive enough? Here’s a round-up of the M&A activity we saw in 2019:


  • Bristol-Myers Squibb acquires Celgene for $74 billion
  • Roche acquires Spark Therapeutics for $4.8 billion
  • Biogen acquires Nightstar Therapeutics for $877 million
  • Pfizer acquires option to buy Vivet Therapeutics for the equivalent of $635.8 million
  • Bayer acquires remaining shares BlueRock Tx for $240m- $360 in milestones with a total $1 billion value

This will be the conversation of 2020. Advanced therapies have the potential to save lives, but this scientific progress means nothing if patients can’t access them. How can the global healthcare infrastructure adapt to ensure we get these life-saving therapies to patients and that reimbursement and access are in sync with approvals?

Zolgensma is a case in point. IT comes with a $2.1 million price tag but it “is a life or death drug”, as Susan put it in her heartfelt speech on patient access, where 68% of children must be dosed by the age of two. Susan went on to explain that while we have the capability to screen in utero, in the US, only 8-10 states are actually screening. In the absence of screening, diagnosis can only be made as the child develops and shows signs of missing certain milestones. With that context, two years becomes an extremely short diagnosis period indeed.

The patient advocate voice is getting louder and organisations like the North Carolina Rare Disease Network are scrutinising state processes and raising awareness amongst politicians and regulators. As an industry we must support these patient advocates!

Let’s take a look at the top ten events in 2019 in summary: 

  1. Patent infringement upheld, Gilead liable
  2. First CRISPR rare disease patients in the US dosed
  3. Pharma and biotech secure manufacturing capacity
  4. J&J/Legend data at ASH
  5. Vertex acquires Semma
  6. Astellas purchases Audentes and Xyphos
  7. Significant CDMO M&A
  8. Licensing deals take centre stage
  9. More fuel for the big pharma fire
  10. Patient access and advocacy 

How does this compare to last year’s top ten; what has changed and progressed, if anything?

Advanced Therapies Week

2022, Miami, Florida.
Book Today