| | | |   | Good morning, everyone. Damian here with a word of caution about CRISPR, a long-awaited drug approval, and the tail of the SPAC boom. | | | Why genome editing is getting harder While the earliest applications for genome editing have largely led to stunning progress, there’s a growing sense among researchers that the next era of CRISPR might not go so smoothly.
As STAT’s Jason Mast reports, broadening the benefits of genome editing will require more complex biological machinery, better targeting, and new methods of delivery. Diseases including cystic fibrosis and HIV, which could theoretically be cured by CRISPR, have proved vexing, leading scientists through a process of trial and error with novel ideas.
“I think it would be surprising if there weren’t some setbacks or some mixed results” with the next generation of genome editing, said Matthew Porteus, who runs a gene-editing lab at Stanford University.
Read more. | After a lengthy delay, Fennec wins FDA approval Fennec Pharmaceuticals won FDA approval yesterday for a drug that reduces the risk of hearing loss in pediatric cancer patients who are treated with cisplatin-based chemotherapy.
The small specialty drugmaker, based in Research Triangle Park, N.C., had assembled all the clinical data needed to secure marketing clearance in 2020, but a series of manufacturing snafus caused two years of delays. The drug will be sold under the brand name Pedmark.
Cisplatin is a commonly used chemotherapy that is highly effective, even curative, for children diagnosed with a variety of solid tumors. But cisplatin is also extremely toxic to the inner ear, so many young cancer patients end up with profound and permanent hearing loss. In clinical trials, the use of Pedmark was found to reduce the risk of hearing loss by about 50% when given to patients after cisplatin chemotherapy. | In-depth analysis of biopharma and the life sciences We may be living through the greatest period of innovation in the history of healthcare. The industry’s efforts to rapidly develop vaccines for Covid-19 is just the latest example. While the industry loves true innovations — and the brilliant minds who envision them — being an innovator is not for the faint of heart. Companies that fall outside the norm face greater scrutiny — even outright pessimism — from various stakeholders. We call this the “Innovator’s Burden.” | Biotech bargains can still be found As biotech’s downturn nears its first anniversary, more than 100 companies are now worth less than the cash they have in the bank. That is at once an indictment of their potential and a windfall-in-waiting for anyone who can convince them to sell.
Take, for instance, Rocket Pharmaceuticals, a gene therapy company that yesterday signed a deal to acquire the flagging Renovacor, which has lost about 80% of its value in the past 12 months. Under the deal, Rocket is trading about $50 million of its own stock for Renovacor’s preclinical gene therapies and, perhaps more useful, the company’s $38 million in cash. That alone will extend Rocket’s runway into the second half of 2024, the company said.
The growing number of biotech companies trading below cash creates the opportunity for larger companies to improve their balance sheets without the risk of selling stock into a bear market. In July, Ginkgo Bioworks functionally raised $337 million in cash in exchange for $300 million in stock by merging with struggling rival Zymergen. | Twilight of the SPAC When the recent blank-check boom began in 2020, there was an implied expiration date: The vast majority of SPACs had two years to find a merger target before having no choice but to return cash to investors. Now, in the back half of 2022, more and more of those firms are calling it quits.
The latest come from investor and former Facebook executive Chamath Palihapitiya, dubbed the SPAC king for having founded a handful of blank-check companies, including the ones that took Akili Interactive, ProKidney, and Clover Health public. In a blog post yesterday, Palihapitiya said he was winding down two SPACs that raised a combined $1.6 billion after failing to find desirable targets.
The news comes two months after Bill Ackman made a similar declaration of defeat on the largest-ever SPAC, returning roughly $4 billion to investors. According to Bloomberg, the index of companies that have gone public through SPACs has fallen more than 80% from its peak in early 2021, with about a quarter of those firms trading at below $2 a share. | More reads - To stem overdoses, Canada is offering safer opioids. Advocates want the U.S. to do the same, STAT
- A chronic disease still waiting for a drug, Wall Street Journal
- FDA user fee lapse would ‘devastate’ public health, Califf says, Bloomberg
| Thanks for reading! Until tomorrow,  | | | |
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