In this issue, we will review the applications of PCR analysis for your gene therapy programs, and present two case studies.

Click to read this audiobook: https://www.altasciences.com/sites/default/files/2023-10/the-altascientist-issue-37-pcr_4.pdf 

Gene therapy continues to accelerate through preclinical and clinical research arenas. These programs are developed with targeted and personalized medicines in mind. The goal of gene therapy is to safely deliver and incorporate a genetic alteration to restore or repair the protein of a missing or faulty gene. Preclinical assessments of gene therapies consider the general absorption, distribution, metabolism, and excretion of the drug, as well as data from tailored assessments to evaluate delivery and cell incorporation. 

Gene therapies require, by design and definition, DNA and/or RNA delivery and analysis. While most ongoing research involves gene therapies being delivered in vivo via adeno-associated viral (AAV) vectors, new in vivo delivery mechanisms are on the rise, such as other types of delivery vectors including lipid nanoparticles.

CHAPTESR: 
- 0:05 — About Issue 37
- 1:20 — Introduction to qPCR, dPCR, and RT-PCR 
- 5:58 — Utility of qPCR, ddPCR, and RT-PCR 
- 8:19 — PCR Applications 
- 10:01 — Advantages and Applications 
- 17:45 — Case Study 1 
- 20:20 — Case Study 2 
- 22:47 — Conclusion

About Altasciences:

Altasciences is an integrated drug development solution company offering pharmaceutical and biotechnology companies a proven, flexible approach to preclinical and clinical pharmacology studies, including formulation, manufacturing, and analytical services. For over 25 years, Altasciences has been partnering with sponsors to help support educated, faster, and more complete early drug development decisions. Altasciences’ integrated, full-service solutions include preclinical safety testing, clinical pharmacology and proof of concept, bioanalysis, program management, medical writing, biostatistics, clinical monitoring, and data management, all customizable to specific sponsor requirements. Altasciences helps sponsors get better drugs to the people who need them, faster.

Issue 37 — Quantitative PCR and Droplet Digital: Analysis for Your Gene Therapy Programs

According to the World Health Organization, at least a billion people are currently living with vision impairment from a preventable or treatable source. The main conditions causing their distance vision impairment or blindness are cataracts, refractive error, age-related macular degeneration, glaucoma, diabetic retinopathy, and presbyopia. 

Precedence Research reports that the global ophthalmic drug market size is estimated to double in 10 years, from $34.6 billion in 2021 to an estimated $68.93 billion by 2030. The development of pharmacological interventions for many of these conditions has been accelerating, supported by new delivery methods and formulation approaches. For conditions that require surgery or corrective devices, eye drops are an important part of diagnosis, and pre- and post-surgery treatment plans. 

In this issue of The Altascientist, we explore how to meet the challenges of formulation development for ocular therapies. 


Issue 43 — Advancing Vision Care With Formulation and Manufacture of Ocular Therapeutics

In this issue of The Altascientist, we review strategies, including those implemented at Altasciences, for developing, qualifying, and implementing CRAs to assess the safety of test articles in vitro to complement in vivo preclinical safety assessment.

Issue 44 — The Importance of Cytokine Release Assays to Derisk the Development of Immunomodulatory Drugs

In this issue, we explore the characteristics and advantages of non-GLP discovery studies, along with emerging trends in this important early phase of drug development research.

We also present two case studies from our Sacramento site, highlighting discovery studies that assessed the biodistribution of the test article under specific experimental conditions.

Issue 45 — Discovery Studies: An Agile Approach to Early Data for Quicker Decisions

In this issue, we examine spray-dried dispersions (SDDs) as an effective method to improve solubility and bioavailability of your active pharmaceutical ingredient (API) in early-phase drug development. We review the spray drying
process, which converts a liquid into a dry powder, and how the resulting SDDs benefit from improved characteristics for further development. We also discuss the combination of spray drying with nanomilling for further bioavailability improvements and flexible downstream processing.

Additionally, we explore the applicability of testing proof-of-concept, as well as creating material for first-in-human (FIH) clinical trials. Leveraging our in-house preclinical services, we also have the unique capability to test spray-dried material versus raw API in vivo, generating meaningful data to compare with in vitro test results.

Issue 46 — Spray-Dried Dispersions

As an end-to-end CRO/CDMO, we have expert knowledge in all early stages of drug development, and we’re sharing that knowledge with you in The Altascientist: The Audiobook. 

Each issue is a deep dive into a specific area of early-phase drug development, providing insider tips, considerations, and case studies. 


Altasciences is a drug development solution company offering pharmaceutical and biotechnology companies a proven, flexible approach to preclinical and clinical pharmacology studies, including formulation, manufacturing, and analytical services.

Issue 37 — Quantitative PCR and Droplet Digital: Analysis for Your Gene Therapy Programs

The Altascientist

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