ramarketing review: Highlights, insights & Spotlights – June

Bringing you insight from our in-house sector experts, the ramarketing review offers vital industry knowledge to keep you in the know, featuring a different ramarketing expert each month to deliver headline highlights.

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Introducing June’s sector expert…

Dr Shona Richardson Ph.D.

Shona recently joined our growing content team as an accomplished Ph.D. qualified Scientific Content Writer.

After graduating with an Undergraduate Degree in Chemistry, Shona completed a Ph.D. at the University of Edinburgh in Biocatalysis. Utilizing the knowledge gained during her Ph.D., she worked as a PostDoc before joining the ramarketing team. This allowed her to expand her expertise further, working on an interdisciplinary project spanning biotechnology, molecular biology and biochemistry.

Enjoy June’s news roundup featuring Shona’s commentary on the headlines impacting companies operating inside outsourcing

 Cell and gene therapy CDMO services market is on the rise

Outsourcing the development of novel treatments is not a new practice, but the recent transition to increasingly complex therapies, coupled with the demand for production speed, has seen a rise in delegating production to contract development and manufacturing organizations (CDMOs).

Particular demand lies in the CDMO services market for complex modalities such as cell and gene therapies (CGT). These therapies have shown promise for treating chronic diseases, which is reflected in the astounding compound annual growth rate of 17.5% projected for CGT manufacturing services over the next decade. This market is predicted to grow from $5.1 billion in 2022 to $11.5 billion by 2027 [1, 2].

An increasing reliance on CDMOs for the production of essential materials is therefore expected. To expand their market share and establish a strong presence in varied geographic locations, key players are focusing on innovative products, conducting product launches and forming long-term alliances [1].

Cell and gene therapy manufacturing: The next generation of startups

Medicine is becoming more personalized, targeting individual patients and their disease manifestations, and moving away from off-the-shelf, one-size-fits-all approaches. Tailored treatments offer specialized methods for treating many rare or chronic diseases. 

However, the CGT approval rate is slow, with only 31 products having been granted approval to date [3]. Therefore, despite significant advancements in the CGT space, there is a persistent bottleneck. New and emerging biotechs aim to overcome the challenges hindering the development of CGT-based precision medicines, with at least half a dozen companies having launched since 2017 with this intent [4].

Beacon Therapeutics, founded in early 2023, has raised $120 million to develop genetic therapies that target eye diseases leading to blindness [5]. Publicly launched in 2022, Upstream Bio has amounted $200 million in investments to tackle asthma with the experimental UPB-101 drug, currently in phase 1b [6].  

With new biotechs continuing to emerge, novel solutions are helping to drive the CGT space forward, leading to a turning point that will see the number of CGT approvals significantly increase in the coming years, to get closer to achieving the FDA’s goal of 10-20 therapies approved annually by 2025 [7]. 

First Duchenne gene therapy for young children approved by FDA

The most recent FDA-approved gene therapy targets Duchenne muscular dystrophy (DMD) in children. The disease causes severe muscle weakness and is the most prevalent form of muscular dystrophy [8]. Granted accelerated approval, this treatment aims to stimulate the production of Elevidys micro-dystrophin, a shortened version of the dystrophin protein present in healthy muscle cells, which DMD sufferers lack [9].

References 

1. Cell & gene therapy manufacturing services market drivers & opportunities | MarketsandMarkets
2. Cell and gene therapy CDMO services market is on the rise | World Pharma Today
3. Approved cellular and gene therapy products | FDA
4. Cell and gene therapy manufacturing: the next generation of startups | BioPharma Dive
5. Built from biotech parts, Beacon launches with $120M to develop eye gene therapies | BioPharma Dive
6. Upstream Bio raises $200M in fresh funding for asthma drug | BioPharma Dive
7. New policies to advance development of safe and effective cell and gene therapies | FDA
8. First Duchenne gene therapy approved by FDA for young children | BioPharma Dive
9. FDA approves first gene therapy for treatment of certain patients with Duchenne muscular dystrophy | FDA

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