Pipeline of cell and gene therapies expands by 25%

By Ben Hargreaves contact

- Last updated on GMT

(Image: Getty/Design Cells)
(Image: Getty/Design Cells)

Related tags: Cell therapy, Gene therapy, Novartis

During the course of just one year, the US pipeline of cell and gene therapies has developed rapidly, as approvals increase.

Early last year​, it was revealed that there were 289 cell and gene therapies held in the US pipeline, this figure has now increased to 362, according to PhRMA.

The updated figure only includes treatments that are currently going through Phase I-III trials, with this figure estimated​ to double once the preclinical pipeline is taken into account.

The jump from 289 to 362 clinical cell and gene therapy candidates represents a jump of 25% in just one year since the last assessment of the pipeline took place.

A knock-on effect of the burgeoning pipeline has been a significant capacity bottleneck​ experienced by contract development and manufacturing organizations to accommodate the work in the area.

Treatment area focus

One area that stayed the same compared to the previous year’s statistics is the concerted focus on oncology treatments. Again, around half of all clinical candidates (173) were focused on oncology, followed by genetic disorders being the next largest therapy area (34).

In total, a third of the drug candidates (132) are aimed at treating rare diseases, which is defined as a condition affecting fewer than 200,000 people in the US.

Challenges ahead

Thus far, nine cell and gene therapies have been brought to the market over the last few years, but PhRMA suggests that there are further challenges ahead once the current pipeline of products moves closer to approval.

In particular, delivery logistics and the manufacture of these products at scale, something companies are already struggling with​, will need to be addressed through ‘large capital investments’.

Andrew Powaleny, director of public affairs at PhRMA, called for greater flexibility in reimbursement of such products.

He stated that current “outdated federal rules and policies can create uncertainty for manufacturers and may limit the growth and expansion of innovative contracting arrangements.”

Last year​, Novartis’ gene therapy treatment, Zolgensma (onasemnogene abeparvovec), was approved in the US and given a price tag of $2.1m (€1.8m) per patient.

This cost made it the most expensive treatment on the market, though the company moved to create an annualized payment plan that would see the treatment paid for over five years.

As more treatments reach the market these kinds of payment structure may become more common, with a spokesperson for Novartis telling us, at the time, that healthcare systems would have to ‘rethink’ how to manage the cost of such treatments.

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