Pavan Cheruvu, Sio Gene Therapies outgoing CEO

January 31, 2022 04:53 PM ESTUpdated February 1, 06:54 AM

Biotech's very bad start to the year continues as microcap gene therapy player Sio ditches CEO, lead partnership

Kyle Blankenship

In a biotech market hit from top to bottom by dwindling investor sentiment, small firms, in particular, are bearing the brunt of the burden. Now, a tiny gene therapy player is rethinking its direction and cutting away a key partnership to make ends meet.

Sio Gene Therapies has elevated CFO David Nassif into the interim CEO role as it says goodbye to current helmsman Pavan Cheruvu, who has left the company to “pursue new opportunities,” the biotech said Monday.

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2023 Spotlight on the Future of Drug Development for Small and Mid-Sized Biotechs

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In the context of today’s global economic environment, there is an increasing need to work smarter, faster and leaner across all facets of the life sciences industry. This is particularly true for small and mid-sized biotech companies, many of which are facing declining valuations and competing for increasingly limited funding to propel their science forward. It is important to recognize that within this framework, many of these smaller companies already find themselves resource-challenged to design and manage clinical studies themselves because they don’t have large teams or in-house experts in navigating the various aspects of the drug development journey. This can be particularly challenging for the most complex and difficult to treat diseases where no previous pathway exists and patients are urgently awaiting breakthroughs.

Christian Itin, Autolus CEO (UKBIO19)

December 9, 2022 06:42 AM EST

Financing

R&D

Cell/Gene Tx

Autolus tips its hand, bags $220M as CAR-T showdown with Gilead looms

Amber Tong

Senior Editor

The first batch of pivotal data on Autolus Therapeutics’ CAR-T is in, and execs are ready to plot a path to market.

With an overall remission rate of 70% at the interim analysis featuring 50 patients, the results set the stage for a BLA filing by the end of 2023, said CEO Christian Itin.

Perhaps more importantly — given that Autolus’ drug, obe-cel, is going after an indication that Gilead’s Tecartus is already approved for — the biotech highlighted “encouraging safety data” in the trial, with a low percentage of patients experiencing severe immune responses.

December 9, 2022 02:03 PM EST

People

Special

WIB22: Amber Salzman had few options when her son was diagnosed with a rare genetic disease. So she created a better one

Nicole DeFeudis

Editor

This profile is part of Endpoints News’ 2022 special report about Women in Biopharma R&D. You can read the full report here.

Amber Salzman’s life changed on a cold, damp day in Paris over tiny plastic cups of lukewarm tea.

She was meeting with Patrick Aubourg, a French neurologist studying adrenoleukodystrophy, or ALD, a rare genetic condition that causes rapid neurological decline in young boys. It’s a sinister disease that often leads to disability or death within just a few years. Salzman’s nephew was diagnosed at just 6 or 7 years old, and died at the age of 12.

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Dipal Doshi, Entrada Therapeutics CEO

December 8, 2022 07:01 AM ESTUpdated 08:58 AM

Deals

Vertex just found the next big ‘transformative’ thing for the pipeline — at a biotech just down the street

John Carroll

Editor & Founder

Back in the summer of 2019, when I was covering Vertex’s executive chairman Jeff Leiden’s plans for the pipeline, I picked up on a distinct focus on myotonic dystrophy Type I, or DM1 — one of what Leiden called “two diseases (with DMD) we’re interested in and we continue to look for those assets.”

Today, Leiden’s successor at the helm of Vertex, CEO Reshma Kewalramani, is plunking down $250 million in cash to go the extra mile on DM1. The lion’s share of that is for the upfront, with a small reserve for equity in a deal that lines Vertex up with a neighbor in Seaport that has been rather quietly going at both of Vertex’s early disease targets with preclinical assets.

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Rami Elghandour, Arcellx CEO

December 9, 2022 11:15 AM ESTUpdated 03:14 PM

Deals

R&D

Updated: Gilead, Arcellx team up on anti-BCMA CAR-T as biotech touts a 100% response rate at #ASH22

Max Gelman

Senior Editor

Gilead and Kite are plunking down big cash to get into the anti-BCMA CAR-T game.

The pair will shell out $225 million in cash upfront and $100 million in equity to Arcellx, Kite announced Friday morning, to develop the biotech’s lead CAR-T program together. Kite will handle commercialization and co-development with Arcellx, and profits in the US will be split 50-50.

Concurrent with the deal, Arcellx revealed its latest cut of data for the program known as CART-ddBCMA, ahead of a full presentation at this weekend’s ASH conference — a 100% response rate among patients getting the therapy. Investors jumped at the dual announcements, sending Arcellx shares $ACLX up more than 25% in Friday’s morning session.

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December 9, 2022 11:09 AM EST

People

Special

WIB22: Leading NK cell researcher reflects on roots in Iran, the UK and Texas

Lei Lei Wu

News Reporter

This profile is part of Endpoints News’ 2022 special report about Women in Biopharma R&D. You can read the full report here.

In a small but widely-cited 11-person study published in NEJM in 2020, seven patients saw signs of their cancer completely go away after getting a new therapy made from natural killer cells. The study was one of the earliest to provide clinical proof that the experimental treatment method had promise.

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December 9, 2022 07:23 AM EST

People

Special