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Umoja and IASO partner to develop therapies for haematological malignancies

Pharmaceutical Technology

The robust expertise of IASO in monitoring possibly best-in-class CARs leveraging its fully-human antibody discovery platform (IMARS), quickly conducting clinical trials and its in-house GMP facility for producing plasmid, virus vector and CAR-T cells intends to make its new therapies available to larger population across the world.

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Editas grabs orphan drug status for sickle cell disease CRISPR therapy

Pharmaceutical Technology

EDIT-301 is made of patient-derived CD34+ haematopoietic stem and progenitor cells, which are edited using CRISPR at the gamma globin gene (HBG1 and HBG2) promoter sites using a proprietary engineered AsCas12a nuclease, per the company’s website. The first patient had a total haemoglobin level of 16.4 g/dL and 45.4%

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Intellia Gets FDA Clearance to Start First Ever Phase III Trial for an In Vivo CRISPR Drug

XTalks

Clinical-stage genome editing company Intellia Therapeutics has received clearance from the US Food and Drug Administration (FDA) for its Investigational New Drug (IND) application to start a pivotal phase III trial of NTLA-2001 for the treatment of transthyretin (ATTR) amyloidosis with cardiomyopathy.

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Avista and Roche to develop gene therapy vectors for eyes

Pharmaceutical Technology

The alliance plans to leverage the single-cell adeno-associated virus (AAV) engineering (scAAVengr) platform technology of Avista to develop intravitreal AAV capsids for a Roche-defined profile. This aids in quickly advancing transformative gene therapies to clinical trials for ophthalmic ailments. Avista is entitled to receive $7.5m

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ESMO 2022: the evolution of TIL therapy – a highly promising approach

Pharmaceutical Technology

As an autologous cell therapy, manufacturing the infusion product is a highly specialised process involving tumour harvest and the extraction and large-scale ex vivo expansion of TILs. The use of engineered immune cytokines that selectively induce the expansion of TILs is being investigated as an alternative to IL-2 stimulation.

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Gene editing: beyond the hype

pharmaphorum

California-based Sangamo Therapeutics is one such company that believes in the powerful potential of in vivo genome editing and regulation, together known as genome engineering, and has built up a sizable preclinical pipeline of genome regulation treatments for diseases such as Huntington’s disease and Amyotrophic lateral sclerosis (ALS).

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RayzeBio raises $160m to advance cancer radiopharmaceuticals

Pharmaceutical Technology

The company is currently progressing its lead drug candidate, RYZ101, into clinical trials for various solid tumour indications. A Phase Ib clinical trial of the drug is underway, enrolling subjects with neuroendocrine tumours, and Phase III trials are anticipated to commence next year.

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