GMP Small Molecules

Stem cell therapies are beginning to enter the clinic, and with the clinical phases of development being highly regulated, there is a requirement for cGMP (Current Good Manufacturing Practice) ancillary reagents (raw materials) to assure therapy safety and suitability. At Tocris we support this development from the research to clinical phase, by offering the very first cGMP small molecules for stem cell therapy. Partner with Tocris today and let us, help you develop the next stem cell therapy.

Targets

GMP Quality

The quality of stem cell therapies and other ATMPs (Advanced Therapy Medicinal Products) depend on the quality and safety of raw materials / ancillary reagents. It is best practice for these to adhere to cGMP specifications when being used for the development of stem cell therapies and other ATMPs. The Tocris range of cGMP small molecules is manufactured following relevant sections of ICH Q7 guidelines (Good Manufacturing Practice Guide for Active Pharmaceutical Ingrediants) guidelines, as well as recommendations from USP <1043> tier 2 risk classification.

Our highly qualified team have full control over the cGMP process, meaning we can ensure from the outset:

  • Batch-to-batch consistency
  • Traceability from starting material to final product
  • Animal-free production

Safety

Safety considerations are of high importance when developing stem cell therapies and other ATMPs for use in the clinic. Our GMP small molecules are synthesized following recommendations from USP <1043> tier 2 risk classification:

USP <1043> : Ancillary Materials for Cell, Gene, and Tissue-Engineered Products

AM Risk Tier 2: Low-Risk, Well Characterized Materials with Intended use as AMs, Produced in Compliance with cGMPs.

To ensure the safety of your stem cell therapy, we can ensure:

  • Traceability from starting materials to final product
  • Fully qualified vendors & raw materials for our production processes
  • Animal-free production
  • Endotoxin and bioburden tested compounds
  • Stringent QC analytical testing (>99% pure by HPLC)

Security of Supply

It is vital to have a consistent and reliable supply of cGMP raw materials when developing ATMPs such as stem cell therapies. Any delay or quality issue can result in significant issues, including increased cost and risk to patient samples. At Tocris we are well known for our consistent and reliable resupply and can offer you:

  • Consistent and assured supply of GMP materials
  • Supply agreements and batch reservations
  • A point of contact at all times throughout your development process
  • Ability to scale up

Why not contact us today to discuss a supply agreement, reserve your batch, and let us know your scale of manufacturing requirements.

Stringent QA and QC

To ensure the highest quality our cGMP compounds are synthesized under a quality assured manufacturing system and subjected to stringent quality control.

Analytical Tests:

  • Purity - validated HPLC (>99% purity)
  • Product identification - NMR and Mass Spec
  • Water content - Karl Fisher
  • Inorganic impurities - Residue on Ignition
  • Residual solvents
  • Metal analysis

Biological Tests:

  • Bioburden
  • Endotoxin

Product Specific Documentation:

  • Certificate of Analysis (CofA)
  • Safety Data Sheet (SDS)
  • TSE/BSE statements (Transmissible Spongiform Encephalopathy / Bovine Spongiform Encephalopathy)

Transition from Pre-clinical to ATMP

An important consideration when developing an ATMP is when to transition raw materials from research use only (RUO) to GMP. Although the quality and safety of raw materials is less important during the pre-clinical stages of development, the cost and freedom of change increases and decreases respectively, as the clinical phase of development is advanced. We would recommend identifying a reliable supplier of your required GMP raw materials early in the preclinical development stage, and transitioning from RUO to GMP as early as possible during development. This will avoid the need to conduct time-consuming and expensive comparative studies during the latter stages of clinical development.

Regulatory Support

We offer the following regulatory support on request for our GMP product line:

  • Auditing of our manufacturing facility (we are based in Bristol, UK)
  • Change notifications
  • Additional product data

FAQs:

What is the difference between RUO and GMP small molecules?

Our cGMP small molecules are manufactured following relevant sections of ICH Q7 guidelines. They are also categorized in the risk tier 2 classification (low risk, well characterized materials with intended use as AMs, produced in compliance with cGMP) of USP <1043>. Our RUO products, along with the RUO products of other suppliers, are categorized in the risk tier 3 classification (moderate-risk materials not intended for use as AMs) of USP <1043>.

We also carry out micro-organism testing on our GMP small molecules, including endotoxin and bioburden testing.

Are GMP small molecules animal free?

Yes, our GMP small molecules are animal free

Can GMP small molecules be used as therapeutics?

No, our GMP small molecules should only be used as raw materials / ancillary reagents for further ATMP manufacture, such as stem cell therapies.

Why has Tocris developed a GMP product line?

Stem cell therapies are beginning to enter the clinic. As the clinical phases of cell therapy development are highly regulated, there is now a requirement to use GMP compounds as ancillary reagents, to ensure the safety, suitability and reduced risk of the stem cell therapy being developed. By introducing a GMP product line, Tocris is fulfilling its commitment to provide the life science community with the latest and most relevant reagents available, to make new discoveries possible.