BiosurgerySven

The BioHub - The Avetix Podcast - S2 Ep71 - Nick Manusos - CEO of Kenai Therapeutics Welcome back to another episode of The BioHub, we were delighted to welcome on Nick Manusos, the CEO of Kenai Therapeutics, a pioneering biotech company developing neuron replacement therapies for neurological disorders. Nick’s journey spans decades of innovation and leadership—from senior roles at global pharma giants like Baxter, Takeda, and Abbott, to founding and leading cutting-edge startups like Iota Biosciences and Opsis Therapeutics. Under his guidance, Kenai Therapeutics has secured groundbreaking funding and is advancing life-changing therapies for patients with Parkinson’s disease. He holds an MBA from Northwestern University’s Kellogg School of Management and a BBA from the University of Wisconsin, bringing a rare combination of business acumen and scientific vision to the table. Nick joins us today to share his insights on pioneering stem cell therapies, building biotech startups from the ground up, and what it takes to turn cutting-edge science into real-world solutions.

What does it take to build a #biotech company from the ground up?

Our CEO, Nick Manusos, sat down with The Bio Hub podcast to share lessons for founders from his own career journey, and how Kenai is harnessing #iPSC technology to cure neurological conditions.

Listen here! bit.ly/42aLMSY

I had a great time developing a new special issue on #GeneEditing for Cell & Gene Therapy Insights. I started with a high level overview prior to the REAL experts! Check it out here: www.insights.bio/cell-and-gen...

A well-placed biotech investor says U.S. policies are straining life sciences startups A well-placed biotech investor says U.S. policies are straining life sciences startups — and more disruption is coming.

A well-placed biotech investor says U.S. policies are straining life sciences startups — and more disruption is coming.
www.statnews.com/2025/07/15/b...

The Trump administration disbanded a newborn screening panel. Advocates now face a harder path atient groups face a harder and unpredictable path going state-by-state to boost screening for rare but treatable conditions after the Trump administration disbanded a federal advisory committee on ne...

In Focus: The Trump administration disbanded a federal newborn screening panel, and patients now face a hard and unpredictable path to diagnosis and treatment.

Congrats to @biosurgerysven.bsky.social and cofounders Michelle Boxall and Lee Buckler on launching Advanced Therapies Think Tank - #AT3, a new platform for unfiltered discussions on the advancement of cell & gene therapies. Season 1 starts July 3!

Learn more: www.advancedtherapiesthinktank.com

FDA shakeup finally gets Nicole Verdun; bros, politics & AI to rule? - The Niche Someone at the FDA has placed Nicole Verdun, the Super Director of The Office of Therapeutic Products at the agency, and her deputy, Rachael Anatol, on administrative leave as of yesterday. The agency...

Analysis: FDA shakeup finally gets key leader Nicole Verdun; bros, politics & AI to rule instead of experienced scientists? ipscell.com/2025/06/fda-... RFK Jr. Oz Makary Prasad Bhattacharya are unlikely to be helpful to biotechs in the cell & gene therapy space #crispr #stemcells

Here: Baby KJ at home with his mom Nicole.
This really happened, folks. Hundreds of people from 8 organizations (led by Rebecca Ahrens-nicklas and Kiran Musunuru) came together to make a bespoke gene editor for this kiddo. He was once in the 7th percentile of body weight. Look at him now! ❤️

DOGE set today as their last day at FDA. Instead, they're continuing to get full pay and don't work FDA lawyer and other federal health agency workers remain on paid leave amid Trump administration cuts, awaiting court decisions while searching for new jobs.

endpoints.news/laid-off-fda... Being paid by the taxpayer to not work. Interesting form of government efficiency?

Effects of atidarsagene autotemcel gene therapy on peripheral nerves in late-infantile metachromatic leukodystrophy In a post-hoc analysis, Zambon et al. find that arsa-cel gene therapy improves nerve function in children with pre-symptomatic late-infantile metachromatic

Arsa-cel gene therapy significantly improves nerve conduction velocities in late-infantile metachromatic leukodystrophy patients compared to untreated peers.

by Zambon AA, Rancoita PMV (...) Fumagalli F et 15 al. in Brain #MedSky

👉 get more here

📖 read the article:

Exclusive: Deerfield secures $600M+ for third healthcare venture fund to seed new companies Deerfield Management is back with its third healthcare venture fund, this time securing more than $600 million to create new drug development outfits, medical technology startups and adjacent computat...

Deerfield has raised $600 million for its third venture fund, backing early biotech startups born out of academic science. The goal: turn promising research into real companies, faster.
endpts.com/deerfield-se...

Early bits of in vivo CAR-T human data emerge from biotech trials in China Initial clinical data from Genocury and EsoBiotec shows promise for in vivo CAR-T therapy, with complete remission in lymphoma and myeloma patients without lymphodepletion.

Two in vivo CAR-T trials in China have delivered early human data, the first clinical readouts of a competitive field.
endpts.com/early-in-viv...

Congratulations to Audrey Greenberg and Miguel Forte, our Executive Fellow Mentors, on being named to The Medicine Maker 2025 #PowerList! A well-deserved honor for two inspiring leaders. See the full list here: bit.ly/4m3AGHK

RFK Jr. touts CMS program testing new ways to pay for sickle cell disease treatment HHS Secretary Robert F. Kennedy Jr. said Friday that 35 states had signed up to participate in a new payment model meant to improve access to and lower costs for gene therapies for sickle cell disease...

New announcement from HHS and CMS - Looks promising for patients? endpts.com/rfk-jr-touts...

First time CEO’s rapidly realise just how isolated they are in their role and being able to provide them access to these global experts is so exciting.

Saisei Ventures Launches Executive Fellows Program Aiming to Enhance Japan’s Biotech Leadership Pipeline Saisei Ventures, a leading venture investment firm focused on advancing Japanese biotechnology and therapeutic innovations, today announced the launch of its...

We’re excited to launch the Executive Fellows Program to mentor the next generation of biotech leaders in Japan. Welcome to our inaugural mentors: Jeff Abbey, Oz Azam, Miguel Forte & Audrey Greenberg! Learn more: bit.ly/3G9hOXd

Measles in the US, update
—712 cases, 11% hospitalized, 3 deaths, 97% cases are unvaccinated
—Now in 24 states
—Actual cases must be multiple thousand to align with the number of hospitalizations and deaths reported
www.cdc.gov/measles/data...
www.cbsnews.com/news/measles...

We're in Rome for the Cell & Gene Meeting on the Mediterranean! Join @biosurgerysven.bsky.social as he moderates a panel discussion on the dynamic ATMP investment landscape.

Date: Wednesday, April 16
Time: 10:45 AM
Location: Salone dei Cavalieri, Section 3&4

Full agenda: bit.ly/42kLqs1
#CGMed25

Rare Disease Biotechs Left in a Lurch as Congress Fails To Renew Priority Review Program Congress did not reauthorize the rare pediatric disease priority review program at the end of 2024. Advocates say the ripple effect is already being felt across biopharma.

Is this the end of the coveted PRV scheme for the FDA? www.biospace.com/business/rar...

Biobanking with genetics shapes precision medicine and global health - Nature Reviews Genetics Biobanks help centralize specimen collections, store and disseminate data, and facilitate large-scale analyses. This Review discusses how biobanks facilitate genetics research towards advancing precis...

As my first post on @bsky.app I am happy to share our paper in Nature Reviews Genetics on how biobanking coupling genetic data with phenotypes at scale shapes precision medicine and global health go.nature.com/3AS54C0. Grateful to Scott Gallagher and Anji Musick for the All of Us Research Program.

Woman who had pioneering cancer treatment 18 years ago still in remission Researchers say woman treated for neuroblastoma as a child is longest known survivor after having CAR T-cell therapy

Some good news for a change.... www.theguardian.com/science/2025... #cellandgenetherapy #celltherapy

In another blow to the CGT commercial environment, Pfizer is stopping their Haemophilia B gene therapy sales. Sad news for patients.

Vertex and Verve part ways on their gene editing collaboration.

First child to die from Measles in a decade. Very sad and totally preventable.

‘The greatest propaganda op in history’: Trump’s reshaping of US culture evokes past antidemocratic regimes The president’s full-court press to dominate media and control cultural institutions is straight out of the authoritarian playbook

"It was also the latest salvo by [Trump] and his allies to control language, influence media narratives and reshape cultural institutions in ways that some compare with the Soviet Union ..."
As an ex-Soviet I agree: I feel like I'm Back in the USSR. In a bad way.
www.theguardian.com/us-news/2025...

Weekly reads: stem cell exhaustion, Lineage Cell trial, Iowa AG wins vs. clinics - The Niche Professor discusses the week's regenerative medicine news including research on the idea of stem cell exhaustion and aging.

Weekly #stemcell & regenerative reads: stem cell exhaustion & aging, Lineage Cell trial encouraging news, Iowa AG wins big vs. unproven clinic firms, more on #stemcells
ipscell.com/2025/02/week...

What Trump gets right about Europe The US president raises uncomfortable but urgent questions over Europe's approach to regulation, innovation and defence.

What Trump gets right about Europe - a tech investor’s thoughts sifted.eu/articles/don...

US research funding cuts put the future of US-based excellence in doubt. on.ft.com/3Q99toc

The Foundations of Time An analysis of the Background Pt. 2

thephysicsjournal.substack.com/p/the-founda...

Susie is the queen of sass 💁‍♀️ 😂 #CalvinandHobbes

In addition to salt marshes - as Neil correctly points out - gene editing, which gave us a curative treatment for sickle cell disease, also has its origins in frog embryos (Aaron Klug's work on zinc fingers) and how yeast have "sex" (Jim Haber work on HO).
"Basic" science -> lifesaving medicines.