Roche made a splash with its acquisition of Spark Therapeutics, whose Luxturna, a gene therapy for an inherited form of vision loss, was the first such therapy to win FDA approval. Now the Swiss pharmaceutical giant is looking to bolster its capabilities in liver and central nervous system (CNS) disorder gene therapies and it’s turning … Continue reading “Roche Enlists Dyno to Bring AI to Next-Generation Liver, CNS Gene Therapies”
Category: Philadelphia
Bio Roundup: CRISPR Kudos, Bristol Myers’s Buy, RNAi Alliance & More
It’s October: a time of days growing shorter, leaves changing color, and phone calls in the wee hours that rouse scientists from slumber notifying them they’ve won the highest honor in their fields. This week, the 2020 Nobel Prize in physiology or medicine was awarded to Harvey Alter, Michael Alton, and Charles Rice, whose research … Continue reading “Bio Roundup: CRISPR Kudos, Bristol Myers’s Buy, RNAi Alliance & More”
BIO Launches ‘Biotech Votes’ Campaign to Encourage Registration and Informed Voting
Science is the way out of this pandemic—and scientific breakthroughs are on the ballot on November 3. To help you get ready for Election Day, BIO’s initiative Biotech Votes has resources to help you get registered and get informed. First, make sure you’re registered to vote. Check out this handy form to verify your voter … Continue reading “BIO Launches ‘Biotech Votes’ Campaign to Encourage Registration and Informed Voting”
Bio Roundup: MESO’s Miss, Pfizer’s Cancer Bet, Vaccine Promise & More
Amid the bombast of Tuesday’s presidential debate, viewers caught some discussion about the response to the coronavirus pandemic and efforts to develop a COVID-19 vaccine. In the crosstalk, the president made an unsupported claim that we are weeks away from having vaccines. It’s true that some pharmaceutical companies are already manufacturing their COVID-19 vaccine candidates … Continue reading “Bio Roundup: MESO’s Miss, Pfizer’s Cancer Bet, Vaccine Promise & More”
With Phase 2 Data in Hand, Ovid Eyes Multiple Pivotal Trials for Epilepsy Drug
Ovid Therapeutics and Takeda Pharmaceutical joined forces three years ago to develop a small molecule into a potential epilepsy drug. That research is starting to pay off as the companies now look ahead to pivotal studies testing that compound in several inherited forms of the disorder. On Wednesday, New York-based Ovid (NASDAQ: [[ticker:OVID]]) reported Phase … Continue reading “With Phase 2 Data in Hand, Ovid Eyes Multiple Pivotal Trials for Epilepsy Drug”
Bio Roundup: Grail Acquisition, Libra’s Launch, Tau Trial Results & More
In biotech, you can go home again. Four years ago, Illumina spun out cancer diagnostics startup Grail. This week, the gene sequencing giant announced it is acquiring Grail in an $8 billion cash and stock deal. Grail set out with the goal of developing a diagnostic test that detects cancer from the genetic snippets tumors … Continue reading “Bio Roundup: Grail Acquisition, Libra’s Launch, Tau Trial Results & More”
Xconomy Awards On-Demand ‘Meet the Finalists’ Webcast Series Continues This Week
The 2020 National Xconomy Awards complimentary two-week, 11-part, on-demand Meet the Finalists daily webcast series continues this Tuesday and Wednesday at 11 a.m. ET. Registration is complimentary and includes access to a networking app with already more than 600 life science contacts. The daily series leads up to the Thursday, Sept. 24 free online National Xconomy Awards … Continue reading “Xconomy Awards On-Demand ‘Meet the Finalists’ Webcast Series Continues This Week”
Bio Roundup: SeaGen Scores, Dyne’s Debut, Microbiome Moves & More
Gilead Sciences CEO Daniel O’Day has spoken openly about acquisitions as a way to build up the company’s drug pipeline. This week, it made its biggest buy yet. The $21 billion Immunomedics acquisition brings to Gilead a cancer drug that many analysts project will become a blockbuster seller. Some of those analysts think Gilead might … Continue reading “Bio Roundup: SeaGen Scores, Dyne’s Debut, Microbiome Moves & More”
Moderna CMO Talks COVID-19 Vaccine at the Xcelerate Keynote Series
The Xcelerate Keynote Series has added Moderna CMO Tal Zaks to the expert lineup of speakers set to present next Monday, Sept. 21. As you likely know, Moderna is one of the front-runners in the COVID-19 vaccine race, and we look forward to adding his insight to a packed agenda focused on innovative solutions during … Continue reading “Moderna CMO Talks COVID-19 Vaccine at the Xcelerate Keynote Series”
Join Xconomy Next Week at the Virtual Xcelerate Keynote Series at Biotech Week Boston
Presented by Xconomy, the online Xcelerate Keynote Series at Biotech Week Boston on Monday, Sept. 21 places a spotlight on life science leaders who are breaking new ground with methods and solutions in R&D, planning, and production that are improving patient outcomes. As the industry navigates uncertainty, the keynote speakers will help drive your business … Continue reading “Join Xconomy Next Week at the Virtual Xcelerate Keynote Series at Biotech Week Boston”
Bio Roundup: COVID-19 Pact, Gavreto Approval, Generative Biology & More
COVID-19 research efforts are often characterized as a race between companies, a description that reflects the urgency to develop a vaccine for the novel coronavirus. The description is not quite right. A race has one winner and public health officials have said we’ll need multiple vaccines to address the global need. Vaccine developers have progressed … Continue reading “Bio Roundup: COVID-19 Pact, Gavreto Approval, Generative Biology & More”
Bio Roundup: Pharma M&A, AbbVie’s Cancer Bet, Biofourmis Bucks & More
Labor Day is just around the corner and many people are looking forward to a last chance to celebrate the waning days of summer. But ahead of the holiday weekend, biopharmaceutical companies were hard at work closing deals. Food conglomerate Nestlé is delving deeper into pharmaceuticals with its $2.6 billion deal to acquire Aimmune Therapeutics, … Continue reading “Bio Roundup: Pharma M&A, AbbVie’s Cancer Bet, Biofourmis Bucks & More”
Early Bird Sale for Xcelerate at Biotech Week Boston Ends This Friday
Don’t miss out on savings for this year’s online Xcelerate Keynote Series at Biotech Week Boston – our early bird sale ends this Friday! Speakers include: Jeff Baker – Deputy Director, Office of Biotechnology Products, FDA Michelle McMurry-Heath – President & CEO, BIO John Maraganore – CEO, Alnylam Pharmaceuticals Yvonne Greenstreet – Chief Operating Officer, Alnylam Pharmaceuticals Andrew Plump … Continue reading “Early Bird Sale for Xcelerate at Biotech Week Boston Ends This Friday”
Biotech Roundup: FDA Culpa, Tricida Trips, Freenome’s Funding & More
Among the casualties of the COVID-19 pandemic: the credibility of government agencies entrusted with protecting public health. The FDA this week granted emergency use authorization for convalescent plasma as a treatment for infection by the novel coronavirus. FDA Commissioner Stephen Hahn and Department of Health and Human Services Secretary Alez Azar both misrepresented the magnitude … Continue reading “Biotech Roundup: FDA Culpa, Tricida Trips, Freenome’s Funding & More”
FDA Adds Plasma as 2nd COVID-19 Treatment Under Emergency Guidelines
The FDA has authorized the use of plasma from people who have recovered from infection by the novel coronavirus as a treatment for hospitalized COVID-19 patients, a move widely perceived as influenced by political pressure given the lack of clear evidence of clinical benefit. Treating patients with plasma—the water, salts, and proteins that make up … Continue reading “FDA Adds Plasma as 2nd COVID-19 Treatment Under Emergency Guidelines”
Astellas Reports Third Patient Death in Audentes Gene Therapy Study
A third patient who received an experimental Audentes gene therapy for a rare neuromuscular disorder has died, a disclosure that comes two months after the deaths of two patients led the FDA to place a clinical hold on the study. San Francisco-based Audentes, a subsidiary of Japanese pharmaceutical company Astellas Pharma, said Friday that early … Continue reading “Astellas Reports Third Patient Death in Audentes Gene Therapy Study”
Bio Roundup: J&J Buyout, BioMarin Rebuffed, Poseida on Hold & More
While business is historically slow in August, this month seems to be bucking the trend, just like so many other unexpected developments in 2020. Perhaps life sciences executives have been less distracted by summer excursions this year in a world in which the COVID-19 pandemic has significantly curtailed travel. This week two pharmaceutical companies announced … Continue reading “Bio Roundup: J&J Buyout, BioMarin Rebuffed, Poseida on Hold & More”
Harmony Bio and Inhibrx Raise Combined $247M in Public Market Debuts
Two biotechs this week topped the $100 million mark in their respective initial public offerings, both of which were able to offer more shares than planned. One, a company with nearly nine month’s-worth of revenue from a newly commercialized drug and the other, an early-clinical stage oncology drug developer, illustrate the range of biotech companies … Continue reading “Harmony Bio and Inhibrx Raise Combined $247M in Public Market Debuts”
Bio Roundup: Seres Soars, MesoBlast Vote, Duchenne Decision & More
The road of drug development is rife with detours and disappointments but Seres Therapeutics showed this week that course corrections can work. Four years after its microbiome therapy failed in a mid-stage study, the company reported early positive data from a pivotal test of its treatment for a type of gut infection. The results are … Continue reading “Bio Roundup: Seres Soars, MesoBlast Vote, Duchenne Decision & More”
Bio Roundup: A Telehealth Combo, FDA’s BCMA OK, Biogen’s Bet & More
The novel coronavirus has driven the pharmaceutical industry to upend its research priorities. Big Pharma companies are focusing on vaccine development and a slew of biotechs are testing compounds, previously intended for a wide range of indications, as potential COVID-19 treatments. This week saw another major development brought on by industry efforts to adjust to … Continue reading “Bio Roundup: A Telehealth Combo, FDA’s BCMA OK, Biogen’s Bet & More”
Bio Roundup: Cerevel’s Shell, Thrive’s Rise, Drug Price Drama & More
The biotech IPO market is buzzing but Cerevel Therapeutics is showing that there’s more than one path to the public markets. This week the neuroscience drug developer reached a deal to merge with publicly traded Arya Sciences Acquisition Corp II. If you’ve never heard of Arya (NASDAQ: [[ticker:ARYBU]]), it’s a shell company that Perceptive Advisors … Continue reading “Bio Roundup: Cerevel’s Shell, Thrive’s Rise, Drug Price Drama & More”
Bio Roundup: Jazz Drug OK’d, Gilead’s Bet, Moderna Patent Decision & More
EQRx launched in January during the JP Morgan Healthcare Conference with an audacious pledge to create 10 new drugs in as many years and then sell them at “dramatically” lower prices than existing medicines. But it offered few specifics about how it would achieve those goals. This week, drug licensing deals it made with two … Continue reading “Bio Roundup: Jazz Drug OK’d, Gilead’s Bet, Moderna Patent Decision & More”
Jazz Pharma Gets FDA Nod for New Drug to Treat Narcolepsy Symptoms
[Corrected 7/23/20, 10:13 am. See below.] Jazz Pharmaceuticals has received FDA approval for a narcolepsy drug that it has developed as a lower-sodium successor to its biggest moneymaker, a treatment that was first approved in 2002 to reduce attacks of muscle weakness associated with the disease. That drug, sodium oxybate (Xyrem), was first OK’d to reduce … Continue reading “Jazz Pharma Gets FDA Nod for New Drug to Treat Narcolepsy Symptoms”
Bio Roundup: Moderna Publishes, Life Science Cash, AdComm Votes & More
If it seems like biotech investments have defied the financial headwinds of the pandemic, maybe it’s because they have. In the second quarter, 16 of the top 25 largest early-stage venture capital deals were investments in life science companies. The figures come from the National Venture Capital Association and financial research firm Pitchbook. This week … Continue reading “Bio Roundup: Moderna Publishes, Life Science Cash, AdComm Votes & More”
Last Chance for Xconomy Awards Nominations – Deadline is Friday, July 17
Have you had your say yet? The 2020 National Xconomy Awards represent the very best our life science industry has to offer. It is our privilege to recognize emerging companies and technologies, highlight groundbreaking business collaborations and strategies, celebrate corporate responsibility and diversity, and promote the innovation that delivers new therapeutics and better health to all. Finalists … Continue reading “Last Chance for Xconomy Awards Nominations – Deadline is Friday, July 17”
Bio Roundup: Blackstone’s Billions, Biogen Finishes Filing, Novavax at “Warp Speed” & More
Cash flowed throughout the life sciences ecosystem this week as Blackstone (NYSE: [[ticker:BX]]) raised a record-setting life sciences fund, companies of all stages announced new financings, and money continued to find its way to those working on efforts to treat or prevent COVID-19. Financiers poured $100 million or more into at least three biotechs, a … Continue reading “Bio Roundup: Blackstone’s Billions, Biogen Finishes Filing, Novavax at “Warp Speed” & More”
NFlection Looks to Erase NF1 Tumors With Drug That Only Goes Skin Deep
The first drug for neurofibromatosis type 1 (NF1) is available to patients following the FDA’s approval of the AstraZeneca treatment earlier this year. Biotech startup NFlection Therapeutics is vying to be runner-up, but its founders say they can eventually win out with a drug that takes a different approach than the commercialized pill while also … Continue reading “NFlection Looks to Erase NF1 Tumors With Drug That Only Goes Skin Deep”
Novavax Gets $1.6B in Operation Warp Speed Funding for COVID-19 Vaccine
The federal government’s Operation Warp Speed has made its biggest financing award yet, promising $1.6 billion to a Maryland biotech to help advance its investigational COVID-19 vaccine and ready millions of doses in preparation for its possible success. Gaithersburg, MD-based Novavax, which has never before brought a product to market, announced the federal award Tuesday. … Continue reading “Novavax Gets $1.6B in Operation Warp Speed Funding for COVID-19 Vaccine”
PacBio CEO, CFO Plan Retirements and More People on the Move
Here’s a look back at some of the life science industry’s executive appointments, departures, and retirements from recent weeks: —Pacific Biosciences (NASDAQ: [[ticker:PACB]]) CEO Michael Hunkapiller and Chief Financial Officer Susan Barnes are retiring. —Moderna (NASDAQ: [[ticker:MRNA]]) appointed Ray Jordan as chief corporate affairs officer. —Aridis Pharmaceuticals (NASDAQ: [[ticker:ARDS]]) appointed Hasan Jafri its chief medical officer. … Continue reading “PacBio CEO, CFO Plan Retirements and More People on the Move”
Bio Roundup: Gilead’s Pionyr Grab, Relay to IPO, Unicorn Sana & More
Uncertainty related to the continued spread of the pandemic continued to consume most of the oxygen in the room this week, but life sciences news outside of that arena continued apace. Once considered all but shuttered, the IPO market again saw an active week between new public debuts and more companies joining the queue. Indeed, … Continue reading “Bio Roundup: Gilead’s Pionyr Grab, Relay to IPO, Unicorn Sana & More”
CSL Behring Bolsters Hemophilia Lineup With Deal for uniQure’s Gene Therapy
CSL Behring is joining the chase for a genetic treatment for hemophilia by acquiring a uniQure gene therapy that’s already in late-stage testing. King of Prussia, PA-based CSL Behring is paying $450 million up front to acquire the uniQure (NASDAQ: [[ticker:QURE]]) asset, etranacogene dezaparvovec (“EtranaDez” for short), the companies announced late Wednesday. Netherlands-based uniQure will … Continue reading “CSL Behring Bolsters Hemophilia Lineup With Deal for uniQure’s Gene Therapy”
Timing Is an Essential Element of Cell & Gene Therapy Product Development
The science underpinning the latest investigational cell and gene therapies is complex enough. But the rapid advance of technologies that support development of these kinds of drugs presents logistical considerations, too. “Drug development is an interesting process because it takes five to 10 years to get a drug to market and invariably, somewhere along that … Continue reading “Timing Is an Essential Element of Cell & Gene Therapy Product Development”
Bio Roundup: A Royal IPO, Akili Arrives, Orca Bio Surfaces & More
The biggest life sciences deal this week involves a company that doesn’t discover or develop drugs, or even sell them. Yet its mark is found on pharmaceutical products spanning the entire sector. Royalty Pharma (NASDAQ: [[ticker:RPRX]]) acquires the royalty rights for drugs. Most of those royalties are for products that are already approved, but the … Continue reading “Bio Roundup: A Royal IPO, Akili Arrives, Orca Bio Surfaces & More”
Pharma Partnering: Perspectives from Xcelerating Life Sciences Panel
At some point, every biotech weighs whether to forge ahead alone or team up with a larger company that has the scientific know-how, clinical trial experience, or the cash to keep a program going. For early-stage developers of cell and gene therapies, these alliances are almost essential. Bruce Levine been on both sides of this … Continue reading “Pharma Partnering: Perspectives from Xcelerating Life Sciences Panel”
Jazz Pharma Gets Quick FDA OK for New Small Cell Lung Cancer Drug
The FDA on Monday approved a new drug for patients with advanced small cell lung cancer, permitting the treatment to be marketed based on less evidence than it traditionally requires in order to speed its path to market. Madrid-based PharmaMar, which developed lurbinectedin (Zepzelca), licensed its US commercialization rights to Jazz Pharmaceuticals (NASDAQ: [[ticker:JAZZ]]) last … Continue reading “Jazz Pharma Gets Quick FDA OK for New Small Cell Lung Cancer Drug”
FDA Reverses Authorization for Use of Antimalarials to Treat COVID-19
The FDA on Monday revoked its emergency authorization permitting use of the antimalarial drugs hydroxychloroquine and chloroquine to treat the novel coronavirus, citing a lack of evidence of their efficacy. In late March the agency authorized the drugs, which President Donald Trump touts often as a potential treatment, for patients who were hospitalized with COVID-19 … Continue reading “FDA Reverses Authorization for Use of Antimalarials to Treat COVID-19”
GlaxoSmithKline Keeps Oncology 2.0 Growth Plans on Track
[Corrected 6/17/20, 1:48 pm PT. See below.] GlaxoSmithKline’s years-long oncology resurgence is on track to have two new drugs approved in the US in mid-2020, after the company won a key first-line ovarian cancer indication for Zejula (niraparib) in April. The belantamab mafodotin and dostarlimab approvals also anticipated this year will give GSK its first … Continue reading “GlaxoSmithKline Keeps Oncology 2.0 Growth Plans on Track”
Biotech Roundup: BIO on Racism, AbbVie Antibodies, IPO Surge & More
At the start of 2020 thousands of biotechies anticipated spending the second week of June in San Diego, hobnobbing at the annual conference hosted by the Biotechnology Innovation Organization. Instead, attendees from 64 countries spanning 28 times zones convened online this week for the first-ever virtual BIO International Convention. There, topics once as unanticipated as the … Continue reading “Biotech Roundup: BIO on Racism, AbbVie Antibodies, IPO Surge & More”
Moderna Finalizes Plan, Dose for Phase 3 Test of mRNA COVID Vaccine
Less than a month after reporting preliminary early-stage data for its experimental vaccine for the novel coronavirus, Moderna has finalized the plan for a Phase 3 test. The study, set to begin next month, is targeting enrollment of about 30,000 volunteers, Moderna (NASDAQ: [[ticker:MRNA]]) announced Thursday. Those patients will be randomly assigned to receive either … Continue reading “Moderna Finalizes Plan, Dose for Phase 3 Test of mRNA COVID Vaccine”
Bio Roundup: Race and Pharma, ASCO Recap, Warp Speed Ahead & More
Race is not a topic that often comes up in pharmaceutical discussions but perhaps it should. Disease doesn’t discriminate by race, but health and economic disparities put some groups at higher risk than others. And yet clinical trials fail to reflect the broad spectrum of patients that drug makers aim to treat. Racial disparities are … Continue reading “Bio Roundup: Race and Pharma, ASCO Recap, Warp Speed Ahead & More”
Cullinan Oncology Reels In $98M to Advance Cancer Drug Pipeline
Cullinan Oncology emerged nearly three years ago with an approach to cancer drugs its founders said would improve the odds of success. Now it has a pipeline of seven compounds and a fresh $98.5 million to advance all of them. The new cash announced Thursday is a Series B round of funding that added the … Continue reading “Cullinan Oncology Reels In $98M to Advance Cancer Drug Pipeline”
SwanBio Therapeutics Appoints Zelenkofske Chief Medical Officer
Gene therapy developer SwanBio Therapeutics has appointed Steven Zelenkofske to serve as its chief medical officer, the same role he held most recently at Achillion Pharmaceuticals, which was acquired by Alexion Pharmaceuticals (NASDAQ: [[ticker:ALXN]]) last year. His experience also includes positions at uniQure (NASDAQ: [[ticker:QURE]]), Regado Biosciences, AstraZeneca (NYSE: [[ticker:AZN]]), Sanofi-Aventis, Boston Scientific (NYSE: [[ticker:BSX]]), … Continue reading “SwanBio Therapeutics Appoints Zelenkofske Chief Medical Officer”
Bio Roundup: ASCO Opens, Merck Takes On COVID, Evofem’s Approval & More
A year ago about 40,000 people gathered in Chicago for the annual meeting of the American Society of Clinical Oncology. Today a virtual version of the ASCO conference, the cancer field’s largest, kicks off, with oncologists and others tuning in from throughout the country and around the world to check out the latest in cancer … Continue reading “Bio Roundup: ASCO Opens, Merck Takes On COVID, Evofem’s Approval & More”
New Webinars: In Vitro Hepatotoxicity Assessment Solutions & Enzyme Fragment Complementation Tech
Xconomy Insight is excited to offer two special webinars to our community this June. Sponsored by Eurofins, registration is free to all. Applying Cell-based In Vitro Assays to Improve In Vitro Hepatotoxicity Assessment Challenges Date: Wednesday, June 10, 2020 Time: 9:00am PDT / 12:00pm EDT Duration: 1 hour Register today In this webinar, we will … Continue reading “New Webinars: In Vitro Hepatotoxicity Assessment Solutions & Enzyme Fragment Complementation Tech”
Generation Bio Leads a Trio of Biotech Companies Aiming for the Nasdaq
COVID-19 has ravaged the economy, and it was expected to quash the IPO market, too. But the biotech sector is defying the pandemic with crossover financings and freshly minted public companies. On Friday, three firms added their names to the list of life science companies preparing to join the public markets. Gene therapy company Generation … Continue reading “Generation Bio Leads a Trio of Biotech Companies Aiming for the Nasdaq”
Bio Roundup: mRNA Vaccine Data, Rise of Rallybio, Roche’s Protein Play & More
As the nation started to reopen this week following two months of closed businesses and stay-at-home orders, public health officials and economists cautioned that society won’t truly return to anything resembling a pre-pandemic normal without a COVID-19 vaccine. Moderna (NASDAQ: [[ticker:MRNA]]) moved one step closer to filling that need with the release of preliminary Phase … Continue reading “Bio Roundup: mRNA Vaccine Data, Rise of Rallybio, Roche’s Protein Play & More”
Bio Roundup: FDA Dings BMS, Genfit’s Fall, Alexion’s Activist & More
This was a disappointing week for at least two major drug makers. French firm Genfit reported a trial failure in an intensely competitive indication, and New York-based Bristol Myers Squibb encountered a delay when the FDA declined to review its experimental multiple myeloma treatment. Genfit (NASDAQ: [[ticker:GNFT]]) was developing a treatment for nonalcoholic steatohepatitis, or … Continue reading “Bio Roundup: FDA Dings BMS, Genfit’s Fall, Alexion’s Activist & More”
Passage Bio’s IPO Raises $216M to Fund Gene Therapy Studies
Gene therapy developer Passage Bio priced its initial public offering at $18 per share, raising $216 million—72 percent more than it originally planned to raise. Earlier this month Passage filed to offer 7.4 million shares, but later increased that to 10 million, then 12 million, shares that sold at the high end of its $16 … Continue reading “Passage Bio’s IPO Raises $216M to Fund Gene Therapy Studies”
Passage Bio Plans IPO as Three Gene Therapies Approach the Clinic
A year ago Passage Bio launched with $115.5 million and plans to advance a handful of gene therapies discovered at the University of Pennsylvania. Now the Philadelphia-based biotech is preparing to join the public markets and raise millions more to move its three lead programs into the clinic. Want more cell and gene therapy content? … Continue reading “Passage Bio Plans IPO as Three Gene Therapies Approach the Clinic”
Passage Bio Gets $116M As Wilson, Yamada Team Up On Gene Therapy Again
Longtime biopharmaceutical executive Tachi Yamada helped keep the work of gene therapy pioneer James Wilson alive 20 years ago when Wilson was mired in controversy and the emerging field was in its darkest days. Now that gene therapy has come of age, in part because of Wilson’s work, the two are teaming up again. They’ve … Continue reading “Passage Bio Gets $116M As Wilson, Yamada Team Up On Gene Therapy Again”