Chi­nese in­vestors back Refuge Bio’s $25M B round as it steers gene en­gi­neer­ing tech to an IND

A group of Chi­nese in­vestors led by 3SBio and Se­quoia Chi­na are bankrolling the next leg of de­vel­op­ment at Refuge Biotech­nolo­gies, a Bay Area biotech that’s been us­ing CRISPR tech­nol­o­gy to con­trol gene ex­pres­sion.

Stan­ley Qi

The syn­di­cate is pro­vid­ing $25 mil­lion in a Se­ries B as Refuge fine tunes a tech­nol­o­gy ini­tial­ly de­vel­oped in the lab of Stan­ley Qi at Stan­ford. Qi mu­tat­ed the Cas9 pro­tein so it couldn’t do the cut­ting used in gene edit­ing. That way it can be used to de­liv­er a tran­scrip­tion­al ac­ti­va­tor or re­pres­sor to turn a gene off or on.

“We don’t seek to com­pete against CAR-T,” he tells me. “We make every­body’s CAR-T that much bet­ter.”

Bing Wang

CEO Bing Wang says that us­ing the tech, Refuge can si­mul­ta­ne­ous­ly tack­le a range of, say, check­point in­hibitors like PD-1 or CT­LA-4 or LAG-3. And he ex­pects the biotech, which had ear­li­er raised $9.5 mil­lion in seed cash and Se­ries A mon­ey, is at least a year and a half away from the clin­ic.

The two co-lead in­vestors have a first right of re­fusal for the Chi­na li­cense to the tech, but he’s free to work with part­ners in the rest of the world.

Francesco Mar­in­co­la

The new round gets Refuge right up to the IND stage, adds Wang, a for­mer in­vest­ment banker at Bar­clays — where he was di­rec­tor of health­care in­vest­ment bank­ing — who says flu­en­cy in Man­darin helped line up the syn­di­cate at a time Chi­nese VCs have been ac­tive­ly seek­ing out US biotechs to in­vest in.

In ad­di­tion to the co-leads new in­vestors Dan­hua Cap­i­tal, San­gel Cap­i­tal and Ocean Pine Health­care Fund jumped in. Refuge’s ex­ist­ing in­vestors, 3E Bioven­tures, WuXi Health­care Ven­tures, and Shang­Bay Cap­i­tal, al­so par­tic­i­pat­ed.

Zhen­ping Zhu of 3SBio and Tren­cy Gu of Se­quoia Chi­na are jump­ing on­to the board of di­rec­tors. And along with the round Refuge an­nounced the ar­rival of Francesco Mar­in­co­la, a vet­er­an of the NIH and Ab­b­Vie, as the biotech’s new chief sci­en­tif­ic of­fi­cer.

Op­ti­miz­ing Cell and Gene Ther­a­py De­vel­op­ment and Pro­duc­tion: How Tech­nol­o­gy Providers Like Corn­ing Life Sci­ences are Spurring In­no­va­tion

Remarkable advances in cell and gene therapy over the last decade offer unprecedented therapeutic promise and bring new hope for many patients facing diseases once thought incurable. However, for cell and gene therapies to reach their full potential, researchers, manufacturers, life science companies, and academics will need to work together to solve the significant challenges facing the industry.

Pfiz­er, Sarep­ta and two oth­ers sug­gest Duchenne drug safe­ty is­sues tied to "class ef­fect"

Since the first experimental Duchenne gene therapy programs came about, the space has proven rife with safety issues and patient deaths in clinical trials. Pfizer and three biotechs now think they’ve found a reason why.

The four companies suggested there may be a “class effect” causing the adverse events in Duchenne gene therapies, they wrote in a new study. They specifically highlighted how side effects in five patients across three trials, who all showed muscle weakness with cardiac involvement, were “strikingly similar.”

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Pre­sent­ing a live End­points News event: Man­ag­ing a biotech in tur­bu­lent times

Biotech is one of the smartest, best educated industries on the planet. PhDs abound. We’ve had a long enough track record to see a new generation of savvy, experienced execs coming together to run startups.

And in these times, they are being tested as never before.

Biotech is going through quite a rough patch right now. For 2 years, practically anyone with a decent resume and some half-baked ideas on biotech could start a company and get it funded. The pandemic made it easy in many ways to pull off an IPO, with traditional road shows shut down in exchange for a series of quick Zoom meetings. Generalist investors flocked as the numbers raised soared into the stratosphere.

Locus CEO and co-founder Paul Garofolo

Lo­cus pulls in a mod­est Se­ries B as it at­tempts to em­u­late on­col­o­gy's pre­ci­sion med­i­cine mod­el in an­tibac­te­ri­als

Despite a significant bear market affecting the public biotech sector, private companies are still managing to pull in VC-backed funding rounds. The latest such round comes out of North Carolina’s research triangle for a precision antibacterial drug maker.

Locus Biosciences closed a $35 million Series B round Wednesday morning, featuring some notable names including Artis Ventures, Tencent Holdings, Viking Global Investors, Discovery Innovations and Johnson and Johnson Innovation, the investment arm of J&J. CEO and co-founder Paul Garofolo told Endpoints News that the funds will be used to advance the company’s lead candidate, dubbed LBP-EC01.

Martin Shkreli (Dennis Van Tine/MediaPunch/IPX)

In­fa­mous biotech ex­ec Mar­tin Shkre­li gets out of prison, hits the street

Martin Shkreli, the infamous biotech CEO who made headlines for his jeering assault on a legion of critics in and out of Congress, is back on the streets after 4 years inside a federal penitentiary.

Shkreli’s attorney put out a statement Wednesday afternoon saying that the “pharma bro” had been transferred to a halfway house in New York with a few more months to go under federal custody, slated to end September 14. Attorney Benjamin Brafman acknowledged the release and vowed that he and Shkreli are keeping quiet.

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De­spite fed­er­al ef­forts to di­ver­si­fy clin­i­cal tri­als, progress re­mains 'stag­nan­t' — re­port

While calls to diversify clinical trials have grown louder in recent years — gaining support from federal agencies such as the FDA and NIH — progress has largely stalled, according to a new report from the National Academies of Sciences, Engineering and Medicine.

Swaths of patients in racial and ethnic minority groups, as well as LGBTQIA+, pregnant and older adult populations continue to be left out of clinical trials. While some advances have been made in the last 30 years — women now account for roughly half of clinical trial participants — growth in other areas remains stagnant, according to the report, which was mandated by Congress and sponsored by the NIH.

Paul Chaplin, Bavarian Nordic president and CEO

Bavar­i­an Nordic se­cures BAR­DA con­tract for small­pox vac­cine

It seems that smallpox vaccination production is weighing on the mind of the US government. And manufacturer Bavarian Nordic is the latest company to benefit.

Just a few days after Emergent, a company that has made government contracts its lifeblood, acquired the exclusive rights to Tembexa from Chimerix, with a $225 million cash payment and an expected BARDA contract, the agency has offered a contract for smallpox vaccine production.

Frank Pallone (D-NJ), House Energy and Commerce Committee chair (Kevin Dietsch/Pool via AP Images)

House com­mit­tee unan­i­mous­ly ad­vances FDA user fee leg­is­la­tion with ac­cel­er­at­ed ap­proval tweaks

The House Energy and Commerce Committee on Wednesday offered a rare show of bipartisan support for a bill that would provide the FDA with user fees for the next five years.

The committee voted 55-0 to advance the quinquennial user fee bill to the full House floor, which if approved, will allow the FDA to use biopharma funds to hire new reviewers, and hit new marks as outlined in the user fee deals that the FDA and biopharma companies forged over the past several years.

Lina Khan, FTC chair (Saul Loeb/Pool via AP)

New FTC com­mis­sion­er could turn the tide for an in­ves­ti­ga­tion in­to PBMs

The Senate last week voted along party lines, 51-50, with Vice President Kamala Harris casting the tie-breaker, to make President Biden appointee Alvaro Bedoya the deciding vote on a split 2-2 Federal Trade Commission.

The addition of Bedoya to the FTC could not only spell more trouble for biopharma M&A activity, as he may align with his Democrat partners to break the FTC ties, but it may also mean that FTC Chair Lina Khan has what she needs to move forward on a study around the pharma middlemen known as pharmacy benefit managers.