CRISPR revolution in cell and gene therapy

This paid-for advertorial by Synthego appeared in Volume 23 – Issue 4, Fall 2022.

Rise of CRISPR-mediated Therapies

After more than two decades of successes and failures, the promise of cell and gene therapy to treat the root cause of many human diseases is now starting to redefine modern medicine. CRISPR genome engineering technology is at the forefront of transforming next-generation cell and gene therapies and personalized medicine.

Re-engineered viruses and non-viral vectors, which are used to deliver therapies, have mitigated some of the adverse reactions experienced in earlier studies. Patients receiving these therapies are experiencing greater benefit with fewer adverse reactions.

A lot of this progress has been enabled by the precision genome editing tool CRISPR. “Saying CRISPR has revolutionized gene therapy is not hyperbole,” says Scott Burger, MD, principal for Advanced Cell and Gene Therapy, a consulting firm specializing in cell and gene therapy product development. “Most gene therapy has focused on gene replacement until recently. CRISPR allows one to potentially approach gene therapy from the standpoint of editing: correcting mutations that cause disease.”

In recent years, CRISPR has shown great potential for developing safer and more effective CAR T-cell therapies, sickle cell disease treatment, as well as targeted therapies for other genetic and non-genetic diseases. CRISPR gene-editing technology may solve problems that have plagued cell and gene therapy development since its inception, as well as issues that have sprouted during the recent cell and gene therapy boom. However, teaming with the right partner is important for ensuring successful process development and safe translation of therapies in the clinic.

Expedite Your Discovery-to-Clinic Journey

Synthego is your trusted partner from discovery to the clinic phase of your CRISPR-based therapy development. With our wealth of CRISPR expertise, proven track record of more than 60 successful GMP campaigns, extensive testing and stability studies, and expert quality team to aid in IND filing of your gene-modified cell therapy, Synthego can ensure that your transition to the clinic is seamless and rapid.

Synthego offers industry-leading, high-quality synthetic sgRNAs in three different grades—research grade (RG), GMP-like, and GMP sgRNA—to support every step of your process.

Large-Scale RUO sgRNA: Large-scale research-grade synthetic sgRNA produced with Synthego’s platform production process under non-GMP conditions.

GMP-Like sgRNA: Synthetic sgRNA that undergo the same manufacturing processes as GMP but with limited documentation.

GMP sgRNA: Clinical-grade sgRNA manufactured under GMP-certified conditions with complete QA/QC oversight and documentation.

Synthego’s State-of-the-Art GMP Facility

Synthego is committed to providing high-quality clinical grade sgRNA that meet or exceed customer and regulatory requirements. Our GMP-compliant facility, located in Silicon Valley, encompasses a quality manufacturing system meeting all required standards:

  • ISO 9001: 2015 certification
  • Compliance with ICH Q7, Section 19, ICH Q9, ICH Q10
  • ISO 7 and 8 clean rooms
  • Packout in ISO 5 Class II BSC
  • Single-pass, HEPA filtered air system
  • Temperature, relative humidity, and differential pressure monitoring
  • Proven track record
  • Segregated manufacturing space

Learn More at Synthego.com/DDW-GMP 

Figure 1. Timeline showing increasing popularity of CRISPR-based cell and gene therapies over the years.
Figure 2: Synthego’s continuum of different sgRNA grades: RUO, GMP-like, and GMP.

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