香港六合彩

IDEA:

Target Product Profile (TPP) 鈥� captures the 鈥榖enefit鈥� of the proposed therapeutic and key considerations in the strategic development of the product (incl. technical, scientific and medical information required to satisfy key stakeholders (regulators and funders)).

Although advanced therapies can include viral and non-viral gene delivery, direct and ex vivo gene therapy, gene supplementation, gene editing and CAR-T cells, AAV is the predominant viral vector used globally with the greatest potential for gene therapy, especially for monogenic loss-of-function diseases. The viral vector used聽is聽dependent on the project and the desired nature of the therapeutic. For example:

香港六合彩 Support:

The Translational Research Group (TRG) have extensive knowledge of the cell and gene therapy landscape, and can provide advice on early considerations such as intended use and competitors etc, to guide development, as well as longer-term considerations for adoption聽i.e. help you produce and develop your TPP.聽

Contact the 香港六合彩 Translational Research Group (TRG)

UNMET CLINICAL NEED:

As part of the TPP, comprehensively research and define the potential for benefit of the proposed therapeutic over current 鈥榮tandard of care鈥�.聽

香港六合彩 Support:聽

The Translational Research Group (TRG) within the Translational Research Office, will help define the project plan to how your project successfully meets the unmet clinical, ensures costs are accurately calculated to cover the project sufficiently, identifies suitable funding streams and helps with the application. Importantly these funding streams require active project management which the TRG can provide throughout the entire project.聽

INTELLECTUAL PROPERTY:

Engage with Business experts聽to ascertain if you have a novel invention, ensure freedom to operate and to protect foreground/arising intellectual property e.g. vector composition.

香港六合彩 Support:

If you believe you have聽an innovative, patentable technology, it鈥檚 important you 聽before any form of public domain disclosure.

香港六合彩B are experts that provide confidential support for researchers at 香港六合彩, to understand the value of your invention and the best approach to protecting your intellectual property (IP),聽advising on strategies to commercialise your research聽such as through forming a spin out company or licensing to a third party.聽

All researchers with potentially commercialisable research results should fill out a confidential Invention Disclosure Form (IDF) and submit it to their 香港六合彩B Business Manager:

ORPHAN DRUG DESIGN:

Sponsors can be granted special status, termed 鈥渙rphan drug designation鈥� (ODD) for a small molecule or biological product (i.e. drug) to treat a rare disease or condition. Such treatments have little commercial incentive but with specific eligibility criteria, ODD brings various development incentives for the novel treatment regarding regulatory support, clinical testing and marketing exclusivity for an orphan drug.聽

Applying for ODD requires submission of data and information by the Sponsor. In Europe, ODD applications are examined by the EMA's Committee for Orphan Medicinal Products (COMP) and in the USA the Food and Drug Administration (FDA).

香港六合彩 Support:

Requiring registration of the Sponsor address in the ODD market region, the Translational Research Group (TRG) within the 香港六合彩 Translational Research Office (TRO), have聽supported the successful ODD of (two) viral gene therapies. 聽

Contact the TRG

PATIENT & PUBLIC INVOLVEMENT (PPI)

Early PPI engagement will help raise awareness of the therapeutic during development, provide opportunities for PPI to shape the development programme, potentially facilitate patient recruitment for subsequent clinical evaluation and support financial investment.

Important PPI considerations:

• Understanding the patient and clinician needs 鈥� Does your TPP meet all of the essential requirements expressed by frontline clinical care providers, patient and public contributors (PPI)?
• Research is carried out 鈥榳ith鈥� or 鈥榖y鈥� members of the public or potential patients etc. 鈥� Advisory members of a project steering group

Further information on PPI can be found via INVOLVE:

香港六合彩 Support:聽

The can help with the timely identification of future needs or opportunities for diversifying, as well as advising on appropriate聽patient groups and charities to consider.聽

Contact the TRG