Intellia Execs Talk CRISPR’s Path Forward at Xconomy’s Oct. 20 Event

Earlier this year, Intellia Therapeutics became the second drugmaker using the landmark gene editing technology CRISPR-Cas9 to go public, after Editas Medicine. A third, CRISPR Therapeutics, could be on the way soon. Now comes the hard part. Getting past the hype and making safe, effective medicines. Over the past few decades, new drugmaking methods like … Continue reading “Intellia Execs Talk CRISPR’s Path Forward at Xconomy’s Oct. 20 Event”

Alnylam Scraps RNAi Drug After Safety Problems, Shares Plunge

Alnylam Pharmaceuticals has gone through more than a decade of twists and turns, trying to become the first company to bring an RNA interference drug, a method of “silencing” disease-causing genes, to market. And while the Cambridge, MA, company still may achieve that goal, it suffered a big setback this afternoon as safety concerns have … Continue reading “Alnylam Scraps RNAi Drug After Safety Problems, Shares Plunge”

Post FDA Approval, Sarepta Wheels and Deals for Combo Duchenne Drugs

Sarepta Therapeutics recently won the first FDA approval for a Duchenne muscular dystrophy drug, eteplirsen (Exondys 51). But the future for patients with the complex, progressive, fatal genetic disease is likely to lie in combinations of therapies, and that’s something Sarepta has clearly decided to pursue through deal-making. For the second time in two weeks, … Continue reading “Post FDA Approval, Sarepta Wheels and Deals for Combo Duchenne Drugs”

Still Stuck Below IPO Price, Merrimack Restructures, CEO Resigns

In the four years since going public, Merrimack Pharmaceuticals has brought its first drug to market, a treatment for pancreatic cancer. But disappointing sales, mounting losses, and a sagging share price have triggered sweeping changes at the Cambridge, MA, company. Merrimack president and CEO Robert Mulroy is resigning, effective immediately, as a result of a … Continue reading “Still Stuck Below IPO Price, Merrimack Restructures, CEO Resigns”

Taking Aim at Alexion, Ra Pharma Heads to IPO Queue

Paroxysmal nocturnal hemoglobinuria may be an ultra-rare blood disease, but Alexion Pharmaceuticals makes over $2 billion a year by selling the only approved drug for it, eculizumab (Soliris). Ra Pharmaceuticals is one of those developing an alternative, and the Cambridge, MA, company is looking to Wall Street to help fund the clinical work. Ra filed … Continue reading “Taking Aim at Alexion, Ra Pharma Heads to IPO Queue”

Bio Roundup: CAR-T & Migraine Races, Intra-Cellular, Rockefeller & More

The presidential race captivated millions of TV viewers this week as the first debate took place in Hofstra. But biotech watchers had other races on their mind. Who will be first to market with a new type of cancer treatment known as CAR-T? Who will break free of the pack of companies developing drugs meant … Continue reading “Bio Roundup: CAR-T & Migraine Races, Intra-Cellular, Rockefeller & More”

Aligned With Roche, SQZ Bio Gets $16M to Push Cell Therapies to Clinic

The first experimental cell-based cancer immunotherapy, known as CAR-T, could soon be submitted to the FDA for approval. But CAR-T therapies have limitations. One of many companies looking to improve them is tiny Boston startup SQZ Biotech, and it just pulled in more cash. The $16 million Series B round SQZ is announcing this morning … Continue reading “Aligned With Roche, SQZ Bio Gets $16M to Push Cell Therapies to Clinic”

Genocea Touts Early Look at Placebo-Controlled Herpes Vaccine Study

To date, the studies Genocea Biosciences has run have not made clear how much better its experimental genital herpes vaccine is than a placebo at treating patients with the infection. Genocea is touting new results today that bolster its case for the vaccine, but which still leave some questions for the company to answer going … Continue reading “Genocea Touts Early Look at Placebo-Controlled Herpes Vaccine Study”

Intra-Cellular Shares Sink As Schizophrenia Drug Flunks Final Test

[Updated 9/29/16, see below] Several promising antipsychotic drugs have made it to late-stage clinical trials only to come up short. Today, you can add an experimental treatment from Intra-Cellular Therapies to the list. Shares of New York-based Intra-Cellular (NASDAQ: [[ticker;ITCI]]) cratered late Wednesday, falling more than 67 percent in after-hours trading after the company disclosed … Continue reading “Intra-Cellular Shares Sink As Schizophrenia Drug Flunks Final Test”

Bayer Exec Dable Takes Reins at Acceleron as Knopf Retires

John Knopf is retiring as the president and CEO of Cambridge, MA-based Acceleron Pharma (NASDAQ: [[ticker:XLRN]]). Knopf, one of the early scientists at the Genetics Institute, co-founded Acceleron in 2003 and took it public in 2013. Bayer veteran Habib Dable, who most recently headed the company’s U.S. pharmaceuticals operations, will take over for Knopf on … Continue reading “Bayer Exec Dable Takes Reins at Acceleron as Knopf Retires”

New Rockefeller Prez Takes Stock of “Wildly Underdeveloped” NY Bio Scene

As the child of a military physicist, Richard Lifton was forced to move around a lot, from Washington, DC, to Albuquerque, New Mexico, to Ankara, Turkey. As an adult he settled in Connecticut, where he became a renowned geneticist at Yale University. Now he’s on the move again, relocating to New York, where he faces … Continue reading “New Rockefeller Prez Takes Stock of “Wildly Underdeveloped” NY Bio Scene”

Neon Therapeutics Taps Novartis Oncology Exec O’Dowd As CEO

Hugh O’Dowd has been named CEO of Cambridge, MA-based immuno-oncology startup Neon Therapeutics, replacing interim CEO and Third Rock Ventures partner Cary Pfeffer. O’Dowd is joining Neon after a more than 20-year stint at Novartis, where he helped commercialize many of the Swiss pharma giant’s cancer drugs, among them imatinib (Gleevec).

Sarepta Preps For Sales As Insurers Unlikely To Deny Duchenne Drug

Kathryn Wagner treats boys with Duchenne muscular dystrophy, a rare and fatal genetic disease. She thinks the first drug ever approved to treat Duchenne is “horrendously expensive,” and she’s not comfortable with the data that the FDA said was good enough to merit the approval, which was announced with great controversy this week. But Wagner … Continue reading “Sarepta Preps For Sales As Insurers Unlikely To Deny Duchenne Drug”

Talk Bio Diversity With Sato, McGuire at Disruptors on Oct. 20

Gender diversity, or rather the lack of it, remains a problem in biotech board rooms, C-suites, and venture firms. As Polaris Partners general partner and co-founder Terry McGuire wrote in a recent op-ed, quite simply, “we need to do better.” McGuire will join Vicki Sato—a professor of management practice at Harvard University and a former … Continue reading “Talk Bio Diversity With Sato, McGuire at Disruptors on Oct. 20”

Eleven Bio Buys Viventia, Rebrands as Cancer Drug Developer

Eleven Biotherapeutics became an eye-disease company several years ago, a move that allowed it to go public, raise a bunch of money, and get an eye drug all the way to a pair of late-stage clinical trials. Both studies failed, however, which left the Cambridge, MA, company searching for a new direction—which today has been … Continue reading “Eleven Bio Buys Viventia, Rebrands as Cancer Drug Developer”

Regeneron, in Pain Drug Race With Pfizer & Lilly, Gets $250M From Teva

Millions of people in the U.S. suffer from chronic forms of pain, but the treatment options aren’t great. Regeneron Pharmaceuticals is locked in a race with Eli Lilly and Pfizer to develop an alternative, and today the Tarrytown, NY, company just got a big chunk of cash to speed the way forward. Teva Pharmaceutical (NYSE: … Continue reading “Regeneron, in Pain Drug Race With Pfizer & Lilly, Gets $250M From Teva”

Sarepta Prices $300K Duchenne Drug As FDA Rift Emerges Over Approval

The FDA approved Monday the first drug ever for Duchenne muscular dystrophy, but only after the commissioner had to settle a high-stakes dispute between powerful agency scientists. Prominent members of the FDA’s drug-review staff, fearful of setting a precedent for the regulation of drugs for rare diseases, voted to reject eteplirsen (Exondys 51), from Cambridge, … Continue reading “Sarepta Prices $300K Duchenne Drug As FDA Rift Emerges Over Approval”

After New Data, FDA Bucks Advisory Panel, Approves Sarepta’s Duchenne Drug

[Updated 9/19/16, 12:42 p.m. See below.] In a decision that could have myriad implications for both the pharmaceutical industry and patients with Duchenne muscular dystrophy, the FDA today approved a drug called eteplirsen from Cambridge, MA-based Sarepta Therapeutics (NASDAQ: [[ticker:SRPT]]). With the FDA nod, eteplirsen has become the first drug ever approved for Duchenne, a … Continue reading “After New Data, FDA Bucks Advisory Panel, Approves Sarepta’s Duchenne Drug”

With $163M Deal, Tarveda Revives Synta Pharma’s Cancer Drug Work

Earlier this year, Blend Therapeutics changed its name to Tarveda Therapeutics and spun what used to be its lead drug into a separate company, the latest steps in a strategic overhaul. The wheeling and dealing continued this morning for the Watertown, MA, company, which has cut a deal with Madrigal Pharmaceuticals—the firm that recently merged … Continue reading “With $163M Deal, Tarveda Revives Synta Pharma’s Cancer Drug Work”

Biotech Roundup: CRISPR’s 3rd IPO, Gender Diversity, Buyouts & More

Across the nation, and even looking back into the past, health was in the headlines this week. Our presidential candidates were either recovering from pneumonia or talking up their health records. New documents came to light that showed the sugar industry paid for studies downplaying the ties between sugar and heart disease. And the drug-price … Continue reading “Biotech Roundup: CRISPR’s 3rd IPO, Gender Diversity, Buyouts & More”

Aerie Shares Rocket As Glaucoma Drug Hits Mark in Phase 3

For people dealing with the creeping blindness of glaucoma, the most effective treatments are eye drops called prostaglandin analogues, or PGAs. They’re not a cure, but taken chronically, they’re the best way to slow the progression of the disease, which is why they’ve been the standard of care for glaucoma for more than two decades. … Continue reading “Aerie Shares Rocket As Glaucoma Drug Hits Mark in Phase 3”

Sofinnova, Atlas Put $35M Into Delinia to Tackle Autoimmune Diseases

Autoimmune diseases occur when our immune systems mistakenly start fighting parts of our own bodies, rather than foreign invaders. The trick is to stop the attack without weakening our body’s defenses. That’s the idea behind a new startup that launched this morning called Delinia. The company, with operations in Cambridge, MA, and San Francisco, CA, … Continue reading “Sofinnova, Atlas Put $35M Into Delinia to Tackle Autoimmune Diseases”

Celgene Touts Data From Early Study of Oral GI Drug

For a few years now, Celgene has been trying to show that the $710 million it shelled out for a gastrointestinal drug from Ireland’s Nogra Pharma was money well spent. Data released this morning from an early study are meant to help boost its case. Celgene (NASDAQ: [[ticker:CELG]]), of Summit, NJ, said this morning that … Continue reading “Celgene Touts Data From Early Study of Oral GI Drug”

Seeking to Join Editas, Intellia, CRISPR Tx Makes Long Awaited IPO Push

Investors hoping to buy into the vast, yet still unproven drugmaking potential of the gene editing system CRISPR-Cas9 will soon have three different companies to choose from. CRISPR Therapeutics filed for an IPO on Friday, meaning it could soon join Boston-area rivals Editas Medicine and Intellia Therapeutics in the expanding club of publicly traded CRISPR-Cas9 … Continue reading “Seeking to Join Editas, Intellia, CRISPR Tx Makes Long Awaited IPO Push”

Pfizer Taps Harvard Professor to Lead Neuro R&D Unit

Pfizer has named Ole Isacson, a neurology professor at Harvard Medical School, the chief scientific officer and senior vice president of its neuroscience research division. Isacson, who has done more than two decades of work in Parkinson’s and other neurodegenerative diseases, will report to Pfizer R&D chief Mikael Dolsten at the pharma giant’s research hub in … Continue reading “Pfizer Taps Harvard Professor to Lead Neuro R&D Unit”

Celgene Plots Speedy FDA Filing for Agios Blood Cancer Drug

It typically takes about 14 years to discover a cancer drug, develop it, and get it to market. Agios Pharmaceuticals has a chance to beat that timeline. Cambridge, MA-based Agios (NASDAQ: [[ticker:AGIO]]) said in a regulatory filing this morning that its partner Celgene (NASDAQ: [[ticker:CELG]]), of Summit, NJ, expects to file for FDA approval of a … Continue reading “Celgene Plots Speedy FDA Filing for Agios Blood Cancer Drug”

With New Name and $47M, Proclara Begins First Big Alzheimer’s Test

Despite a litany of high profile failures among drugs to fight Alzheimer’s disease, biotechs and pharma companies continue to pour billions of dollars into the fight. The latest firm to get a financial boost is a startup from Cambridge, MA, that until today has been known as Neurophage Pharmaceuticals. The company has raised $47 million … Continue reading “With New Name and $47M, Proclara Begins First Big Alzheimer’s Test”

Allergan Dips Toes into Gene Therapy With $60M Retrosense Buy

The attempt to commercialize gene therapy has been a multi-decade roller coaster, but its current upswing continues. Today Allergan, a company best known for skin and eye care products, has acquired a startup developing gene therapies for a range of eye diseases. The deal is pocket change for a company that brought in over $4 … Continue reading “Allergan Dips Toes into Gene Therapy With $60M Retrosense Buy”

Rigel Touts Late-Stage Data for Platelet Drug

Back in 2013, Rigel Pharmaceuticals, having seen a few drugs fail in clinical trials, decided to go after less common health problems rather than the major diseases, including asthma and rheumatoid arthritis, it had before. There’s still more to be done before that decision pays off with Rigel’s first approved drug, but the South San … Continue reading “Rigel Touts Late-Stage Data for Platelet Drug”

Filing: Moderna Blowing Past Record $450M Round With New $600M Raise

Moderna Therapeutics has already raised the largest biotech financing round in history, yet the Cambridge, MA, company looks to be on its way to breaking that record just a year later. According to a regulatory filing, Moderna—the messenger RNA drugmaker that has already vacuumed up more than $1 billion in financing and partnership dollars—is raising … Continue reading “Filing: Moderna Blowing Past Record $450M Round With New $600M Raise”

Bio Roundup: Mylan’s Shock, Hillary’s 3%, Duchenne Lessons & More

It was the tweet heard around the biotech world, again. Mylan bumped up the price of the EpiPen 400 percent, presidential nominee Hillary Clinton responded with a tirade against drug price hikes, and—much like when Clinton railed against drug pricing last year—biotech indexes promptly plummeted more than 3 percent. Last year, Clinton’s target was Martin … Continue reading “Bio Roundup: Mylan’s Shock, Hillary’s 3%, Duchenne Lessons & More”

Duchenne Lessons: Advocates For Rare Spinal Disease Have Eyes on FDA

[Updated, 1:09 pm ET, see below] Heather and Jessica Tomko will tell you that they’re nothing alike. They’ve got different interests, different tastes in movies, television, and music. If Jessica hates a gift, Heather probably loves it. Heather just scored tickets to see the Broadway show Hamilton and can’t wait. Jessica sighs. “You can’t get … Continue reading “Duchenne Lessons: Advocates For Rare Spinal Disease Have Eyes on FDA”

Former Bristol Exec Albright Named CSO of Editas

Cambridge, MA-based gene editing company Editas Medicine (NASDAQ: [[ticker:EDIT]]) has named Charles Albright its chief scientific officer. Albright was most recently the VP of genetically defined diseases and genomics at Bristol-Myers Squibb, and held positions at Incyte and DuPont Pharmaceuticals before that.    

Pfizer Shells Out $14B to Win Medivation Sweepstakes

Pfizer has just won the sweepstakes for San Francisco Bay Area cancer drugmaker Medivation, with a buyout made official early Monday morning. The Financial Times first reported late Sunday that Pfizer (NYSE: [[ticker:PFE]]) was close to finalizing a $14 billion deal for Medivation (NASDAQ: [[ticker:MDVN]]), whose shares closed on Friday at $67.16 apiece. On Monday … Continue reading “Pfizer Shells Out $14B to Win Medivation Sweepstakes”

Cerulean’s Shares Routed as Cancer Drug Comes up Short Again

Cerulean Pharma survived a near-death experience once before. But the Waltham, MA-based company now has another crisis on its hands. Cerulean (NASDAQ: [[ticker:CERU]]) said that its lead drug, CRLX101, failed its primary goal in a Phase 2 trial of 115 patients with renal cell carcinoma, a type of kidney cancer. The drug, when combined with … Continue reading “Cerulean’s Shares Routed as Cancer Drug Comes up Short Again”

CRISPR and More at Boston’s Life Science Disruptors Oct. 20

It wasn’t too long ago that the gene editing system CRISPR-Cas9 was just a tool for research labs, not companies developing drugs. But that’s all changed, and now it’s on companies like Intellia Therapeutics (NASDAQ: [[ticker:NTLA]]) to write the next chapter of what could be one of the most important biotech stories of the century. … Continue reading “CRISPR and More at Boston’s Life Science Disruptors Oct. 20”

As Pfizer Buys, Closes Bind Tx, CEO Hirsch Heads to Agios

The ink is barely dry on Pfizer’s agreement to buy Bind Therapeutics out of bankruptcy and shut it down, but the employee exodus has begun. Andrew Hirsch, who was Cambridge, MA-based Bind’s (NASDAQ: [[ticker:BIND]]) chief financial officer before taking over for Scott Minick as CEO in 2015, has been named the CFO of another Boston-area … Continue reading “As Pfizer Buys, Closes Bind Tx, CEO Hirsch Heads to Agios”

OncoGenex Hires Advisor, Evaluates Options As Cancer Drug Fails Again

It wasn’t too long ago that OncoGenex Pharmaceuticals had high hopes that a drug called custirsen might make a big impact on the fast-moving treatment landscape for prostate cancer. A couple of failed trials later, however, the Bothell, WA, company has now hired a financial advisor to see if it still has a path forward. … Continue reading “OncoGenex Hires Advisor, Evaluates Options As Cancer Drug Fails Again”

Bio Roundup: Precision Med and Google, Heron, Lilly, Sarepta & More

The wait continues. An FDA ruling on a Duchenne muscular dystrophy drug from Sarepta Therapeutics—which could be the first ever approved for the disease—has been imminent for a while now, yet Labor Day is fast approaching and still the saga drags on. Speculation abounds, from analysts and pundits, each giving different opinions as to whether … Continue reading “Bio Roundup: Precision Med and Google, Heron, Lilly, Sarepta & More”

MIT’s Jacks Leans on Disney Family, Not VCs, to Hatch New Startup

Tyler Jacks is a cancer research pioneer, one of the co-chairs of the White House’s ambitious Cancer “Moonshot” initiative, and has helped advise a number of companies developing cancer drugs over the years. But only now is Jacks building a cancer drug maker of his own: a stealthy startup called Dragonfly Therapeutics. Dragonfly was formed … Continue reading “MIT’s Jacks Leans on Disney Family, Not VCs, to Hatch New Startup”

Aldeyra Surges on New Data, Another Step For Inflammation Drug

Step by step, a small biotech called Aldeyra Therapeutics has been trying to build a case for a new type of inflammation-fighting medicine. Data released this morning by the Lexington, MA-based company continue to support the idea. Aldeyra said its drug, a medicine called NS2 meant to “trap” toxic aldehyde molecules, succeeded in a mid-stage … Continue reading “Aldeyra Surges on New Data, Another Step For Inflammation Drug”

Proteostasis Expands C-Suite, Adds Three New Execs

Cambridge, MA-based Proteostasis Therapeutics (NASDAQ: [[ticker:PTI]]) appointed three new members to its executive team. James DeTore (Bluebird Bio’s (NASDAQ: [[ticker:BLUE]]) former chief financial officer) is Proteostasis’s new CFO; Geoffrey Gilmartin (most recently a medical lead at AstraZeneca) is the company’s chief development officer; and Marija Zecevic (founder of consulting firm Zebra Ventures) is heading up … Continue reading “Proteostasis Expands C-Suite, Adds Three New Execs”

Merck Surges As Bristol’s Immunotherapy Drug Stumbles in Lung Cancer

Over the past few years, Bristol-Myers Squibb and Merck have brought to market two rival immunotherapy drugs, so-called checkpoint inhibitors, that have begun to change the way certain cancers are treated. But immunotherapy still has a long way to go, and Bristol provided the latest evidence Friday morning, when its drug, nivolumab (Opdivo), failed a … Continue reading “Merck Surges As Bristol’s Immunotherapy Drug Stumbles in Lung Cancer”

Biotech Roundup: Biogen Rumors, Theranos, Electro-Drugs & More

Biogen was once again all over the biotech newswire this week. First came a positive surprise; data that could set the stage for the first-ever drug approved for spinal muscular atrophy, a rare and debilitating muscle disease. Then came the rumor mill: Companies may or may not be approaching Biogen about a sale, and Biogen … Continue reading “Biotech Roundup: Biogen Rumors, Theranos, Electro-Drugs & More”