Cell & Gene weekly
EU approval for Hemgenix, C&G M&A and more
EU approves gene therapy for hemophilia B
Three months after receiving the US FDA approval, CSL’s candidate Hemgenix has received the green-light from EMA for the treatment of hemophilia B (!)
Now the fun begins with everything related to price and access… As a reminder, Hemgenix's list price in the U.S. is $3.5 M. Even if ICER suggests that this price tag could be ‘justified’, it won’t be an easy one to convince EU payers on - Good luck to CSL !
“Nature is healing”: Three Sickle Cell Disease programs discontinued
There’s a lot (too many?) C&G therapy Sickle Cell Disease programs out there, especially considering the ambition for these treatments to be ‘one and done’ : i.e., time is of the essence to capture the prevalent patient population. A quick search clinicaltrials.gov [link to search] suggests there are >40 clinical trials in this space across academia, biotechs (CRISPR/ Vertex, Editas, Beam, etc.) and big pharma (Novartis/Intellia, Sanofi/Sangamo, CSL, etc.). - yikes -
… now three key contenders have dropped out of the race:
Graphite Bio announced Thursday it was discontinuing the development of nula-cel in SCD. [link]
I’m going to go ahead and say that I called it in my Jan 2023 newsletter edition [link]. - After all, in early Jan, they had voluntarily paused the program after a SAE the 1st patient dosed …
Novartis announced it would no longer develop their Intellia-partnered ex-vivo program OTQ923 [link]
Sanofi announced that it was handing rights back to Sangamo for SAR445136 [link]
This will make it easier for the two front runners, CRISPR / Vertex and Bluebird Bio, who still seem to be on track for a 2023 FDA submission
AbbVie and Capsida Bio expand their collaboration to develop genetic therapies for serious eye diseases
The collaboration will use Capsida's novel adeno-associated virus (AAV) engineering platform and manufacturing capability to identify and advance three programs. The collaboration builds on a 2021 neurodegenerative disease partnership with AbbVie in which the Big Pharma put down $90 million for three CNS disease targets.
Under the expanded agreement, Capsida will receive $70M, consisting of upfront payments and a potential equity investment, and be eligible to get up to $595M in ‘biobucks’, with potential for commercial milestones, and royalties.
The partnership comes one month after the gene therapy platform company signed a deal worth up to $685 million ‘biobucks’ with Eli Lilly.
Source: Fierce Pharma
Moderna's latest genomics move sees it team up with Life Edit to develop new gene editing therapies
With their mountains of cash, Moderna is fueling their investments into Cell & Gene : good ! Moderna have now made a series of pacts / partnerships, cementing their resolve for this space
In September 2021, Moderna formed a pact with Vertex for gene editing CF programs [source]
In November 2021, Moderna signed a pact with Metagenomi for gene editing technologies [source]
In October 2022, Moderna signed a deal with Autolus for proprietary binders [source]
In January 2023, Moderna aquired Oriciro genomics for cell-free synthesis and amplification of plasmid DNA [source]
NOW: Partnership with Life Edit Therapeutics with the aim of developing next-gen gene editing therapies for hard-to-treat diseases. The gene editing platform of Life Edit provides an extensive and diverse library of base editors and RNA-guided nucleases (RGNs), which are smaller in size than conventional nucleases.
Source: Fierce Biotech
Quick take news:
AviadoBio Signs Exclusive License Agreement with Neurgain Technologies for Novel Gene Therapy Spinal Delivery Technology [source]
*Any views and opinions expressed herein are those of the author (Marco Sabatini) and do not necessarily reflect those of his employer