$40M up front: Skyhawk takes flight with Vertex deal

Skyhawk Therapeutics Inc., one of the early leaders in developing small-molecule drugs that target RNA, closed out the year with yet another big pharma partnering deal, an alliance with Vertex Pharmaceuticals Inc., in which it is getting $40 million up front plus up to $2.2 billion more in milestones and royalties on product sales.


A busy, buzzy Vertex finds a high-profile partner in the RNA-drugging world to launch a fresh discovery effort

Vertex is fronting the pact with $40 million in cash to get things started. And then there’s more than $2 billion in milestones, which indicates the size of its ambitions. What we don’t know — as is often the case with collaborations — is exactly what Vertex is trying to do here.


Skyhawk chief Bill Haney will say what the general focus is: neurological.

And he was also impressed that Vertex’s team under Boston site head Mark Bunnage knew exactly what kind of targets they were interested in. From there, using Skyhawk’s bioinformatics tech, Haney says there was a straightforward path to determining the likelihood of success and creating a partnership.


Vertex joins Skyhawk's roster of A-list partners, securing options on RNA splicing modulators

Vertex has secured exclusive options to license Skyhawk Therapeutics’ candidates that modulate RNA splicing. The deal will see Vertex pay Skyhawk $40 million upfront and commit to up to $2.2 billion in milestones to secure options on multiple small molecules. 

Skyhawk, led by Bill Haney, has landed deals with a who’s who of leading biopharma companies on the strength of a platform that generates drugs designed to modify expression of genes once thought to be undruggable. Biogen, Bristol Myers Squibb, Merck, Roche and Takeda have all signed up to work with Skyhawk to access the fruits of the platform.


The Endpoints 11: Here are some of the most promising startups in biotech. And Covid-19 isn't going to stop them.

Bill Haney, an entrepreneur and filmmaker who has a finger in a multitude of pots, got this biotech started with a mere $8 million in seed cash. Since then, he’s brought in hundreds of millions more from marquee partners like Merck who have been convinced that the startup has the key to drugging RNA — one of the hottest plays in the discovery field.


Haney’s other biotech, Dragonfly, was recognized in our first Endpoints 11. And he’s been wheeling and dealing on a blur of deals for both ever since. What made Skyhawk successful, he adds, had a lot to do with Kathleen McCarthy, the co-founder and CSO, and Sergey Paushkin, VP of biology at the biotech. They both had early involvement in the work that helped birth risdiplam, the remarkable SMA drug that helped open the door to this field.


Merck doubles down on Skyhawk's tech to drug RNA, promising $600M per autoimmune, metabolic target

Early collaborations with big-name partners lent Skyhawk Therapeutics both the endorsements and the capital to pursue a small molecule platform for drugging RNA. And they soon wanted more. When Merck signed on to discover candidates against neurodegeneration and cancer last July, Biogen was just expanding the list of neuro targets they want to go after. Now Merck is coming back for its own round two, adding autoimmune and metabolic diseases to the menu.


Skyhawk Therapeutics boosts Merck collab pact with added targets, upfront and biobucks


Merck has decided to double down on its research pact with startup Skyhawk Therapeutics as it expands the deal to now also cover autoimmune and metabolic diseases.


The 20 under 40: Inside the next generation of biopharma leaders

A scientist corrals top talent in her entrepreneurial role. 


U-2 Dragon Lady, one of the conference rooms at Skyhawk Therapeutics' office in Waltham, MA, is named after both the American single-jet engine, high altitude reconnaissance aircraft and founder Kathleen McCarthy.2


Experienced Business Leader in Biopharmaceutical Industry Joins as Chief Operating Officer at Skyhawk Therapeutics

Dr. Terrance Connolly brings over 20 years of biotech operations and business development experience to his role as Chief Operating Officer Skyhawk Therapeutics.


The 20 Startups to Watch in Boston in 2020


Based on factors like funding, partnerships, awards, technology licenses and more, we've worked to identify the startups that are on the precipice of something big. These companies, we think, will be making major moves to kick off the new Roaring 20s. 


Celgene execs shell out $92M cash for a pair of R&D deals that will fit perfectly in their new home at Bristol-Myers


Celgene is shelling out $80 million in a cash upfront to add oncology, immuno-oncology and autoimmune diseases to the initial roundup of neurological targets mapped early in Skyhawk’s existence.


Skyhawk’s $80M Celgene deal expands remit to autoimmune


In Skyhawk’s fifth deal of the year, the company is moving into a new therapeutic area and adding $80 million to its cash reserve. Skyhawk partnered with Celgene to discover and develop small molecules for autoimmune disease and cancer against hard-to-drug targets that have been clinically validated, or genetically associated with a disease.


C&EN’s 2019 10 Start-Ups to Watch


Skyhawk Therapeutics: Drugging RNA splicing with small molecules to treat disease


Fattening the bankroll, Bill Haney adds cancer R&D powerhouse Merck to Skyhawk's roster of partners out to drug RNA


Whatever Biogen learned about Skyhawk in the 6 months since it anted up $74 million to get a collaboration going with their R&D team on drugging RNA for neurodegeneration, it must have been a positive experience.


Bill Haney's Skyhawk spears another marquee alliance for its work drugging RNA in Takeda neuro pact


Bill Haney’s Skyhawk Therapeutics has scored another high-profile development partner.


Top 20 Life Science Startups to Watch in 2019

BioSpace is proud to present its NextGen Bio “Class of 2019,” a list of 20 up-and-coming life science companies in North America that launched* no earlier than 2017.


The NextGen Bio Class of 2019 is a stellar group of companies that are already making an enormous impact on the industry now and will continue into the future. Congratulations to this group!


Joining forces with Skyhawk, C4 Therapeutics, Biogen buys more shots on goal on SMA, Alzheimer’s


These days at Biogen, the spotlight tends to fall on one of two programs: Its commercial SMA drug Spinraza, and the experimental Alzheimer’s therapy aducanumab — and its dealmaking team knows it.


Biogen looks to expand neuro pipeline via C4, Skyhawk deals


A pair of deals announced Friday leaves Biogen Inc. (NASDAQ:BIIB) poised to expand the druggable space of its neurology pipeline. Biogen entered R&D partnerships with targeted protein degradation company C4 Therapeutics Inc. (Cambridge, Mass.) and spliceosome company Skyhawk Therapeutics Inc. (Waltham, Mass.).


Creating Companies Before 35: Meet the Young Innovator Award Finalists


Entrepreneur, scientist, doctor, startup founder, CEO, and in some cases, all of the above. This year’s Young Innovator award finalists—all 35 and under—have shown that they can do it all to try to translate an idea into new treatments....   


[Kathleen McCarthy] started thinking about how small molecules could be used to correct RNA splicing errors... She went to her friend, tech entrepreneur and investor Bill Haney, with the idea for a company, and together they co-founded Skyhawk Therapeutics in 2016, where McCarthy, 34, is chief scientific officer. Skyhawk recently announced a $60 million deal with Celgene (NASDAQ: CELG) and $40 million in financing from a variety of investors.


The new serial entrepreneur in the biotech arena launches another upstart looking to drug RNA


A year after launching the cancer startup Dragonfly — allying himself with MIT’s Tyler Jacks and David Raulet out of Berkeley — he’s now back with his second upstart called Skyhawk Therapeutics, which is jumping into the fast-growing new company segment focused on drugging RNA with small molecules.


Skyhawk, copying Dragonfly model, gets cash for RNA R&D


Skyhawk Therapeutics has raised $8 million to move small molecules that correct RNA expression toward the clinic. The startup draws on the same striking mix of executives and investors that have powered natural killer cell player Dragonfly Therapeutics to a deal with Celgene.


Disney, Family Offices Back Second Bio Startup from Tech Entrepreneur Haney


To launch his first biotech startup, Dragonfly Therapeutics, Bill Haney went to private family offices, including one linked to the Disney family, rather than venture firms. Now the tech investor, entrepreneur and filmmaker is at it again, leaning on some of the same investors to fund Skyhawk Therapeutics, a new company joining an emerging race to target RNA with drugs.


Exon skipping newco Skyhawk launches with $8M


RNA exon skipping drug company Skyhawk Therapeutics Inc. (Waltham, Mass.) debuted Tuesday with $8 million in a seed financing led by Alexandria Venture Investments, family investors Tim Disney and the Duke of Bedford.


With $8M seed round, Waltham biotech puts new twist on ‘exon-skipping’


Unlike Sarepta Therapeutics, which makes a drug that skips over faulty exons — parts of genes that encode proteins; Skyhawk Therapeutics is developing treatments for diseases that are caused by exon-skipping.


Skyhawk Therapeutics, Inc.

35 Gatehouse Drive


Waltham, MA 02451

United States of America


Tel: 617.858.0041

Skyhawk Therapeutics Europe GmbH


Basel Technology Park

Hochbergerstrasse 60C

Basel 4057


Tel: +41 (0) 61 638 23 50

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