Taysha Acquires Rare Disease Gene Therapy For $5.5 M; AZ’s Farxiga Fails In COVID-19 Study; Sanofi To Strengthen Its Vaccines Manufacturing Capacities

Posted on Apr 13, 2021 by Delveinsight

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Taysha takes charge of a Rare Disease Gene Therapy for $5.5 M 

Taysha Gene Therapies announced the acquisition of exclusive global rights to TSHA-120 from a leading patient advocacy group for an upfront payment of USD 5.5 million. 

TSHA-120 is an intrathecally dosed AAV9 gene therapy designed to treat giant axonal neuropathy (GAN). Giant axonal neuropathy is a rare genetic disorder affecting over 2000 people in the US and Europe. It is a result of loss-of-function mutations in the gene coding for gigaxonin. It severely affects the peripheral as well as the central nervous system

TSHA-120 has earlier received rare pediatric disease and orphan drug designations for the treatment of giant axonal neuropathy. The therapy is currently undergoing clinical trial investigation in collaboration with NIH and the patient advocacy group focused on finding treatments and cures for GAN. 

Besides upfront payments, the patient advocacy group is also eligible to receive additional clinical, regulatory and commercial milestones ringing in at up to USD 19.3 million as well as low, single-digit royalties on net sales after the product’s commercialization.

AZ’s Diabetes Drug Farxiga Fails in COVID-19 Study

AstraZeneca has had a rough journey with its COVID-19 vaccines after its use was restricted by European and British regulatory agencies amidst risks of blood clots. Further, the image of the vaccine was marred by the delays in its rollout. 

Besides the vaccine, AZ was also focusing on repurposing existing diabetes drug Farxiga for the treatment of coronavirus infection. Farxiga is a first-in-class, oral, once-daily SGLT2 inhibitor and stands approved to lower down the potential risks of hospital visits for heart failure in patients with type 2 diabetes and established cardiovascular disease or multiple cardiovascular risk factors.

Sadly the drug failed to reach endpoints in a Phase III study as a potential treatment for hospitalized COVID-19 patients serious risk of developing complications. The trial did not achieve statistical significance in the Phase III DARE-19 study after a 30-day period and fell short of expected results.

Sanofi Continues to Strengthen its Vaccines Manufacturing Capacities

Learning from the ongoing pandemic and vaccine crisis, Sanofi has embarked on a journey to maintain a strong network of vaccine manufacturing capacities with the announcement of another expansion in Singapore.  

The construction of the manufacturing unit is expected to begin in the third quarter of the year, and the site will be recognized as the regional centre and main supplier for vaccines in Asia.

The company is prepared to dish out a sum of roughly $476 million over the next five years while bringing cutting-edge technology to the site. The unit will have the potential to produce three to four vaccines simultaneously in comparison to one vaccine at a time at the moment.

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