Altasciences The Altascientist

Explore the key considerations for successful formulation development and manufacture for safety assessment, and learn about the strategies Altasciences employs to support sponsors.

- 0:35 — Introduction
- 3:34 — Considerations for Preclinical Drug Formulation
- 7:36 — Preclinical Drug Product Manufacturing
- 9:46 — Steps in Preclinical Drug Product Manufacturing
- 13:49 — Quality Assurance and Control
- 14:33 — Regulatory Aspects
- 15:42 — Risk Management
- 17:17 — Case Study
- 21:27 — Conclusion

Formulating and manufacturing drugs for preclinical testing is an early, crucial step toward human trials. At the preclinical stage, the goal is to maximize exposure for safety testing, formulating to the limits of solubility and maximum volumes. To optimize the formulation for preclinical testing, one must consider the route of administration, selection of excipients, stability testing, plans and options for scale-up, and contingency plans.

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.

In this issue, we review the key considerations and regulatory guidance for nonclinical cell and gene therapy development, including expert approaches to mitigating complex challenges, improving study efficiency, and maximizing translational opportunities to first-in-human trials.

CHAPTERS:
- 00:07 — About This Audiobook
- 01:57 — Introduction
- 05:34 — Species Selection
- 09:00 — Germline Mitigation Studies
- 13:22 — Planning Your Nonclinical Studies
- 14:44 — Case Study 1
- 17:18 — Case Study 2
- 19:08 — Conclusion

Click here to read Issue 36: https://www.altasciences.com/sites/default/files/2023-09/The-Altascientist-Issue36-Cell-and-Gene-Therapy.pdf

Traditional approaches modulate the course of disease but do not provide a cure, particularly in the case of monogenic diseases, which are caused by mutations in single genes that a person is born with. It is estimated that there are greater than 6,000 monogenic diseases, affecting over 350 million people worldwide. For these diseases, cell and gene therapy may provide hope for a cure.

There are significant challenges associated with the successful development of these complex, leading-edge therapies. Challenges involved in the in vivo preclinical study of cell and gene therapies include understanding on- and off-target activity, immune responses, and other serious adverse events. All of these must be carefully monitored, rigorously assessed, and managed to the highest extent possible.

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.

The demand for HPAPIs has been increasing over the past decade, mainly driven by oncology research. The overall HPAPI market is predicted to reach $31.5 billion USD by 2029, with more than 25% of drugs currently on the market formulated with HPAPIs.

In this issue, we examine the intricacies involved in manufacturing highly potent active pharmaceutical ingredients, herein referred to as HPAPIs. This examination of HPAPIs includes a review of the relevant guidance, classification systems, and safety processes. We also delve into how CDMO facilities, equipment, and processes for HPAPIs should be adapted to maximize safety and success for your development projects.

CHAPTERS:
- 00:08 ― Introduction
- 01:35 ― Defining High Potency for an API
- 03:47 ― Determining Exposure Potential
- 05:47 ― Engineering Controls for HPAPI Manufacturing
- 08:25 ― Regulatory Requirements for HPAPI Production
- 14:22 ― Altasciences Case Study
- 17:25 ― Conclusion

Click here to read Issue 35: https://www.altasciences.com/sites/default/files/2023-08/The-Altascientist_issue35_HPAPI.pdf

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.

Understanding a novel drug's PK and PD properties begins with nonclinical studies and evolves through early phase clinical trials. In this issue, we take a closer look at the collection and use of PK/PD data in early phase drug development.

CHAPTERS:
- 0:08 — Introduction to Issue 34
- 1:32 — Nonclinical Studies
- 2:39 — IND Requirements and Translation to Clinical PK/PD
- 5:37 — Translating Nonclinical Knowledge of PK/PD Analyses to Clinical Study
- 8:51 — Case Study
- 12:32 — Conclusion

Click here to read Issue 34: https://www.altasciences.com/sites/default/files/2023-08/the-altascientist-issue-34-toxicokinetics-pharmacokinetics-and-pharmacodynamics.pdf

Introduction on TK, PK, and PD Data:

The understanding of a new drug’s absorption, distribution, metabolism, and excretion (more commonly known as ADME) is critical to ensure that it is safe for human use. That understanding is achieved through collection and analysis of pharmacokinetic (PK) and pharmacodynamic (PD) data, which together account for approximately 25% of the contents of a drug package insert or label.

The characterization of PK/PD effects starts with nonclinical toxicokinetic (TK) studies in animals. The purpose of TK studies is to define the chemical properties of the drug, including pharmacology and toxicology, and to assist in development of downstream clinical protocols. The necessary nonclinical studies are conducted before submission of Investigational New Drug (also known as IND) applications to the FDA or other global regulatory agencies, and deliver critical data used to set the parameters for future clinical trials.

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.

A consideration with a measurable impact on early phase clinical studies is the location where trials are performed; this is especially true for first-in-human trials. Conducting early phase clinical research on novel compounds in Canada can provide significant advantages versus other locations.

CHAPTERS:
- 0:43 — Introduction
- 3:12 — Canadian Clinical Trials: Time and Cost Savings
- 3:46 — CTA (Health Canada) vs. IND (FDA)
- 5:17 — CTA: Health Canada vs. EMA
- 7:52 — Regulatory Review Process Predictability
- 10:19 — Business Efficiency Cost Savings
- 11:28 — Clinical Trial Participants
- 13:06 — FAQs
- 18:43 — Why Altasciences?

Read along with this PDF version:

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.