Trends Changing Biopharma: C&GTs – redefining treatments and fostering a personalized approach

Written by Shona Richardson, Scientific Content Writer

Cell and gene therapies (C&GTs) are emerging disease treatments with immense potential, and over 1500 of them are currently in clinical trials.¹ Over the past decade, significant investment has led to an impressive average growth rate for cell and gene therapies (63% and 59%, respectively), and this increased interest is expected to continue for the foreseeable future.² Innovation in synthetic biology is driving C&GT advancements in producing life-changing treatments for chronic diseases.

The biopharma industry is changing, and as its dedicated marketing partner — we pride ourselves on staying up to date with developments and trends to fully understand how the sector is evolving.

So what are the trends currently shaping the future of C&GTs?

CRISPR technology and gene therapy

Clustered regularly interspaced short palindromic repeats (CRISPR) is a powerful gene editing tool that can alter DNA by adding, replacing, or neutralizing specific genes. It redefines how we treat certain diseases, and its current targets include blood disorders, AIDS, cystic fibrosis, and cancer.

Alternative gene editing techniques can also be utilized — including zinc finger nucleases or transcription activator-like effector nucleases — but CRISPR remains a popular option for gene manipulation.³ Once genes have been edited, they are administered into the body using systems such as viral vector technology, lipid nanoparticles, or are integrated into cells in combined C&GTs.

CRISPR-mediated gene correction has recently been applied to produce life-altering treatments for hemophilia B.⁴ Hemgenix is the first gene therapy to be granted regulatory approval for the treatment of hemophilia B, illustrating how synthetic biology is driving C&GT innovation.⁵ Whereas previous treatment relied on the repeated transfusion of the protein Factor IX responsible for blood clotting, the new therapy is designed to be effective after a single dose, significantly improving patients’ quality of life by reducing the number of clinic visits required.

CRISPR technology has also been shown as an effective method to engineer T cells to produce CAR-T therapies; these treatments identify and kill cancer cells and comprise 49% of current therapies from preclinical to pre-registration.⁶

As a result, gene editing technology and therapeutics are changing our approach to medicine, curing previously incurable genetic disorders. One drawback is that C&GTs suffer from a very low approval rate — only 14% from phase 2 — which is limiting the number of therapies brought to market. Therefore, the market hinges on overcoming the challenges that are hindering C&GT approvals to bring essential medication to patients.¹

Changing cell therapy landscape: CAR-T and ACT

In cell therapy, modified cells are injected, grafted, or implanted into a patient to provide a beneficial medicinal effect. These cells can be sourced from a donor (allogeneically), or using a patient’s own cells (autologously).

As mentioned above, cell therapy is closely linked to gene therapy, and many treatments utilize combined C&GTs. This is the case for CAR-T treatments, where T cells are extracted from the patient, genetically modified, and re-introduced. Many cell and combined therapies are currently in clinical trials, targeting specific disorders; examples include T-cell receptor solid tumor treatments and allogeneic T-cell therapy.⁷ The FDA recently fast-tracked an allogeneic CAR-T therapy, which is currently in phase I clinical trials for the treatment of relapsed and refractory multiple myeloma.⁸

Research published earlier this year utilized adoptive cell therapy (ACT), which harnesses immune cells collected from a patient’s tumor to treat hard-to-access cancer types, revolutionizing less-invasive alternatives to harsh chemotherapies.⁹

Personalized medicine

Personalized medicine customizes a treatment for a specific individual. This patient-centric approach enables tailor-made therapies, moving away from “one-size-fits-all” solutions. A good example are autologous C&GTs, where the patient’s cells are used, modified, and re-injected to produce a medically beneficial effect. A useful tool for enabling these treatments — CRISPR — has been harnessed to engineer personalized treatments.

Investigations have also identified CRISPR-based diagnostics that could enable home-based disease biomarker identification to diagnose chronic illnesses by target-specific binding.¹⁰ This would widen patient access to diagnostic tools and reduce the load on healthcare workers. An example of this is specific high-sensitivity enzymatic reporter unlocking, known as SHERLOCK-based diagnostics, which works by target amplification followed by CRISPR-mediated sequence detection. During the COVID-19 pandemic, this method detected SARS-CoV-2 in saliva and nasal samples.¹¹⸴¹²

Advancements in C&GT are helping to drive personalized medical approaches and increase patient access to crucial diagnostic capabilities.

Decentralized manufacturing

Decentralized manufacturing divides production across multiple sites or geographic regions. This is especially appealing for C&GTs as cold-chain shipping adds logistic complexity, with requirements for specific storage conditions (below –60 to –150 °C) and limited shelf life. Increasing the number of geographic locations can lower this complexity by considerably reducing the distance from manufacturing to destination.

Investments in C&GTs have tripled in the past five years and manufacturing practices need to become more robust to meet this growing demand.¹³ Therefore, companies have been seen to outsource to CDMOs for local production to maximize utilization and minimize costs. This decentralized approach also supports the development of personalized medicine by increasing patient access.

Impact on the industry

Innovative C&GT treatments are revolutionizing medicine, opening opportunities for treating diseases untouched by current medical therapies in addition to offering more patient-focused customized treatments. As companies optimize their current manufacturing processes and more treatments are approved, C&GTs will likely change the medical field and immeasurably improve the quality of many lives.

Seeking the support of the right marketing specialist can help you communicate your innovative research and expertise effectively, setting you out as a leading expert in C&GTs.

As a specialist life sciences marketing agency, we support companies and to help them get noticed. Our sector experts can help to showcase your unique selling propositions to customers and generate leads. Support your company’s growth contact ramarketing today.

Sources:

1. 2023’s Market Outlook For Cell And Gene Therapies (outsourcedpharma.com)
2. Cell Gene Therapies Investment Outlook In 2022 Beyond (cellandgene.com)
3. Genome Editing Techniques: The Tools That Enable Scientists to Alter the Genetic Code (synthego.com)
4. CRISPR/Cas9-mediated gene correction in hemophilia B patient-derived iPSCs
5. EU approves first gene therapy for hemophilia B (biopharma-reporter.com)
6. Cell, Gene, & RNA Therapy Landscape (asgct.org)
7. 10 Cell and Gene Therapies to Watch in 2023 (cgtlive.com)
8. Caribou gets FDA fast track for allogeneic CAR-T (biopharma-reporter.com)
9. Isolation of tumour-reactive lymphocytes from peripheral blood via microfluidic immunomagnetic cell sorting
10. CRISPR as a Diagnostic Tool
11. CRISPR-based diagnostics
12. Detection of SARS-CoV-2 with SHERLOCK One-Pot Testing
13. Growth in Cell & Gene Therapy predicts a wave of demand for biomanufacturing space (jll.com)