Biogen Strikes Alzheimer’s Deal, Paying $45M for Ionis Tau Drug Rights

When it comes to Alzheimer’s disease, Biogen isn’t putting all of its eggs in the same basket. In the latest development of a multi-drug and multi-disease collaboration, the company has licensed an experimental Ionis Pharmaceuticals compound that works in a different way than the therapies currently in its pipeline for neurodegenerative diseases. On Thursday, Ionis … Continue reading “Biogen Strikes Alzheimer’s Deal, Paying $45M for Ionis Tau Drug Rights”

Takeda Pays Turnstone $120M to Start Alliance on Viral Cancer Therapies

[Updated 12/20/19, 2:51 p.m. See below.] Turnstone Biologics has yet to test its engineered viruses in cancer patients but Takeda Pharmaceutical sees enough promise in the technology to pay $120 million up front to start a partnership developing the biotech’s lead therapeutic candidate. The Takeda (NYSE: [[ticker:TAK]]) payment is a combination of cash, near-term milestones, … Continue reading “Takeda Pays Turnstone $120M to Start Alliance on Viral Cancer Therapies”

Ring Therapeutics Launches to Expand Gene Therapy Viral Vector Options

Ring Therapeutics, a Flagship Pioneering spinout, launched Thursday with ambitious plans to expand the universe of vectors available for gene therapy delivery. Gene therapy, treatments intended to treat disease by inserting a gene instead of using drugs or surgery, has had a banner year, with the second ever such therapy approved this year in the … Continue reading “Ring Therapeutics Launches to Expand Gene Therapy Viral Vector Options”

Zafgen’s Path Forward Is Reverse Merger With Chondrial Therapeutics

Zafgen stumbled in clinical trials testing its experimental therapies for metabolic diseases. Going forward, it will have a new name, new management, and a mitochondrial disorder drug from another company. Boston-based Zafgen (NASDAQ: [[ticker:ZFGN]]) has entered a merger agreement with Chondrial Therapeutics, a rare disease drug developer just entering the clinic. Chondrial shareholders will own … Continue reading “Zafgen’s Path Forward Is Reverse Merger With Chondrial Therapeutics”

Ipsen’s Meek Departs for CEO Role at Gene Therapy NewCo FerGene

Ipsen chief executive David Meek has resigned from the Paris-based pharmaceutical company to join FerGene as its CEO. Cambridge, MA-based FerGene was formed in November by Ferring Pharmaceuticals and Blackstone Life Sciences, which pumped a combined $570 million into the new business. Operating as a subsidiary of Ferring, FerGene will handle US commercialization of nadofaragene … Continue reading “Ipsen’s Meek Departs for CEO Role at Gene Therapy NewCo FerGene”

Nimbus Therapeutics Taps Annie Chen to Lead Subsidiary

Nimbus Therapeutics has named Annie Chen to the role of president at its subsidiary, Nimbus Lakshmi. Chen is currently chief medical officer of Cambridge, MA-based Nimbus Therapeutics, and she will continue serving in that role. The company uses software to discover and design new drugs, some of which are developed by its subsidiaries. Nimbus Lakshmi … Continue reading “Nimbus Therapeutics Taps Annie Chen to Lead Subsidiary”

Triplet Therapeutics Unveils $59M to Try Hairpin Fix for Huntington’s

Huntington’s disease drug research has focused mainly on the genetic roots of the rare neurodegenerative disorder. Triplet Therapeutics is taking a different tack by targeting a DNA repair mechanism gone awry, an approach that CEO Nessan Bermingham says could offer a better way to treat that disease and potentially dozens of others. Cambridge, MA-based Triplet … Continue reading “Triplet Therapeutics Unveils $59M to Try Hairpin Fix for Huntington’s”

Organovo’s Demise Offers Path for Tarveda to Join the Public Markets

Organovo raised millions of dollars from investors to fuel its goal of developing 3D “bioprinted” human tissues for use in people with end-stage liver disease and other life-threatening illnesses. But the San Diego company fell short of bringing its lead regenerative medicine program into clinical trials, and now it is charting a course as a … Continue reading “Organovo’s Demise Offers Path for Tarveda to Join the Public Markets”

Wave Stock Crashes After Decision to Halt Duchenne Drug Studies

Wave Life Sciences saw its stock price plunge nearly 60 percent Monday following the company’s decision to discontinue development of its investigational Duchenne muscular dystrophy drug, suvodirsen, after an interim analysis from a Phase 1 study. Cambridge, MA-based Wave (NASDAQ: [[ticker:WVE]]) was testing suvodirsen as a potential treatment for Duchenne, a genetic neuromuscular disorder that … Continue reading “Wave Stock Crashes After Decision to Halt Duchenne Drug Studies”

Cell Reprogramming Startup Bit Bio Inks Pact With Charles River

Charles River Laboratories International is making moves in the cell therapy space after signing on the cell reprogramming startup Bit Bio and announcing plans to acquire a biomaterials company for $380 million. The Wilmington, MA-based contract research organization (CRO) last week announced that it has entered into an exclusive discovery and safety services partnership with … Continue reading “Cell Reprogramming Startup Bit Bio Inks Pact With Charles River”

Building a Better 3D Bioprinter: GE Healthcare and ASLS Combine Tech

GE Healthcare Life Sciences and ASLS have teamed up to offer a new platform that can design, build, and image living 3D tissue models for drug discovery and development. ASLS, Advanced Solutions Life Sciences, is a subsidiary of Advanced Solutions headquartered in Louisville, KY. Its patented Angiomics technology enables users to create 3D tissue models … Continue reading “Building a Better 3D Bioprinter: GE Healthcare and ASLS Combine Tech”

Pyxis Oncology Names Sullivan CEO, Steinberg Remains on the Board

Pyxis Oncology has appointed Lara Sullivan to serve as its CEO. She is also joining the Boston company’s board of directors. Sullivan succeeds David Steinberg, Pyxis’s founding CEO and a general partner at Longwood Fund. Steinberg will remain on the company’s board as an independent director. Sullivan was most recently founder and president of Pfizer … Continue reading “Pyxis Oncology Names Sullivan CEO, Steinberg Remains on the Board”

Bio Roundup: Sarepta’s Approval, Merck M&A, Drug Price Bill & More

If you’re keeping track, FDA drug approvals have been on a tear in the past month with several decisions coming well ahead of their targeted dates. And then there’s Sarepta Therapeutics. Late Thursday, the FDA announced approval of the Cambridge, MA, biotech’s drug for Duchenne muscular dystrophy in patients who have a specific mutation. The … Continue reading “Bio Roundup: Sarepta’s Approval, Merck M&A, Drug Price Bill & More”

In an About-Face, FDA Approves Second Sarepta Muscular Dystrophy Drug

Sarepta Therapeutics received an early holiday gift Thursday—approval of golodirsen (Vyondys 53), its second Duchenne muscular dystrophy treatment, which the FDA rejected in August. The agency had dinged the application over potential side effects, namely a risk of infections from the “ports” used to infuse the drug, and kidney problems observed in animals in which … Continue reading “In an About-Face, FDA Approves Second Sarepta Muscular Dystrophy Drug”

Iterum’s Antibiotic for Super Bugs Falls Short in Phase 3, Shares Sink

Iterum Therapeutics’ bid to bring patients a new antibiotic that’s safer than currently available drugs has fallen short of the main efficacy goal of a late-stage clinical trial. But the company is pinning its hopes on another Phase 3 study that will report data early next year. Iterum (NASDAQ: [[ticker:ITRM]]) reported preliminary Phase 3 data … Continue reading “Iterum’s Antibiotic for Super Bugs Falls Short in Phase 3, Shares Sink”

Flagship’s Cellarity Aims to Advance Cell Behavior-Based Therapeutics

The latest startup to emerge from Flagship Pioneering aims to change drug discovery by starting the process with a clear understanding of cell behavior. Cellarity—like many other startups looking to revamp the slow, costly process of developing new medicines—is using machine-learning tools to do so, leveraging computational advances to advance a new way of thinking … Continue reading “Flagship’s Cellarity Aims to Advance Cell Behavior-Based Therapeutics”

Flexion Co-Founder, Chief Scientific Officer Neil Bodick to Retire

Neil Bodick, chief scientific officer of Flexion Therapeutics (NASDAQ: [[ticker:FLXN]]), will retire on Jan. 3. After co-founding the company in 2007, Bodick served as its chief medical officer. He became chief scientific officer in 2017 following FDA approval of the company’s extended-release formulation of triamcinolone acetonide (Zilretta), a treatment for knee pain. After retiring, Flexion … Continue reading “Flexion Co-Founder, Chief Scientific Officer Neil Bodick to Retire”

Replimune Taps Ex-Merrimack Exec for Chief Financial Officer Post

Replimune Group (NASDAQ: [[ticker:REPL]]) has appointed Jean Franchi to serve as chief financial officer, the same position she held most recently at Merrimack Pharmaceuticals (NASDAQ: [[ticker:MACK]]). Franchi’s experience also includes positions at Genzyme, Dimension Therapeutics, and Good Start Genetics. Woburn, MA-based Replimune is developing oncolytic immune-gene therapies to treat cancer.

KalVista Pharma Eye Drug Partnered With Merck Flunks Phase 2 Test

A KalVista Pharmaceuticals eye drug at the center of a research collaboration with Merck has failed a mid-stage clinical trial. KalVista (NASDAQ: [[ticker:KALV]]) developed its drug, KVD001, to treat diabetic macular edema (DME), a complication of diabetes that can lead to blindness. On Monday, Cambridge, MA-based KalVista said patients treated with the drug, given as … Continue reading “KalVista Pharma Eye Drug Partnered With Merck Flunks Phase 2 Test”

Perceptive Unveils New $210M Fund to Invest in Early-Stage Biotechs

Perceptive Advisors, an investment firm with a long track record of life science investing across all points of development, is now stepping up to pump money into companies at the earliest stages with a new $210 million venture capital fund. The Perceptive Xontogeny Venture Fund (PXV) is led by Chris Garabedian (pictured above, center), a … Continue reading “Perceptive Unveils New $210M Fund to Invest in Early-Stage Biotechs”

Merck Makes a Big Blood Cancer Bet With $2.7B Deal for ArQule

Merck is acquiring cancer drug developer ArQule for $2.7 billion, a cash deal that brings it a promising early-stage compound that could challenge a blockbuster leukemia drug from AbbVie. According to terms of the agreement announced Monday, a Merck (NYSE: [[ticker:MRK]]) subsidiary will pay $20 for each share of ArQule (NASDAQ: [[ticker:ARQL]]), a 107 percent … Continue reading “Merck Makes a Big Blood Cancer Bet With $2.7B Deal for ArQule”

AstraZeneca Taps Gatehouse Bio to Identify Potential Drug Targets

After joining AstraZeneca’s Boston Bio Hub incubator last year, Gatehouse Bio is now teaming up with the pharma company to identify potential drug targets in respiratory and cardiovascular diseases. Under the collaboration, expected to last multiple years, Gatehouse will use its sRNAlytics platform to identify small RNA (sRNA) signatures, which the company says help in … Continue reading “AstraZeneca Taps Gatehouse Bio to Identify Potential Drug Targets”

3 Takeaways From the Resurrection of Biogen’s Alzheimer’s Drug

The name of the experimental drug was heard so frequently in hotel hallways at the conference that it sounded like an incantation: aducanumab, aducanumab, aducanumab. Regardless of the eventual FDA verdict on Biogen’s once-bagged, now-revived anti-amyloid antibody, data from clinical trials of the treatment have inarguably altered the course of drug discovery and development for … Continue reading “3 Takeaways From the Resurrection of Biogen’s Alzheimer’s Drug”

Akcea Therapeutics Appoints Alex Howarth Chief Operating Officer

Akcea Therapeutics (NASDAQ: [[ticker:AKCA]]) has named Alex Howarth its new chief operating officer. He was most recently president at Lycera. His experience also includes positions at moksha8, Vitae Pharmaceuticals, and GlaxoSmithKline (NYSE: [[ticker:GSK]]). Howarth joins Boston-based Akcea following a corporate shakeup in September that led to the departure of three executives. The rare disease drug … Continue reading “Akcea Therapeutics Appoints Alex Howarth Chief Operating Officer”

FDA Puts Partial Hold on Tests of Ipsen Drug Acquired in $1B Deal

Two clinical trials testing an Ipsen drug in rare connective tissue disorders have been placed on partial hold after safety concerns about chronic dosing of the compound emerged. The partial hold applies to patients under 14 years of age in Phase 2 and Phase 3 tests of the drug, palovarotene. But the Paris-based company, which … Continue reading “FDA Puts Partial Hold on Tests of Ipsen Drug Acquired in $1B Deal”

Bio Roundup: Biogen at CTAD, Sage’s Stumble, Astellas’s $3B Offer & More

It’s been a roller-coaster year for those hoping for new drugs to treat Alzheimer’s disease, a leading cause of death with rising treatment costs and no clear answer yet as to its cause. Clearing proteins, such as amyloid and tau, was once seen as the top contender for stopping or slowing neurodegeneration. Costly failures forced … Continue reading “Bio Roundup: Biogen at CTAD, Sage’s Stumble, Astellas’s $3B Offer & More”

Biogen Points to Enrollment and Dosing to Explain Alzheimer’s Results

Biogen revealed detailed results from its Alzheimer’s trial to a jam-packed room of eager conference attendees this week—and while some described it as a major advance in the field, others were less convinced. Samantha Budd Haeberlein, Biogen’s vice president of clinical development, took the stage Thursday morning in San Diego at Clinical Trials on Alzheimer’s … Continue reading “Biogen Points to Enrollment and Dosing to Explain Alzheimer’s Results”

FDA Rejects Enzyvant’s Regenerative Therapy for Rare Immune Disorder

[Corrected 12:27 p.m. See below.] The FDA has rejected an experimental regenerative therapy that Enzyvant developed to restore immune system function in babies born with a rare disorder. The agency cited manufacturing issues, according to the company. Enzyvant received the notification, formally known as a complete response letter, on Wednesday, CEO Rachelle Jacques tells Xconomy. … Continue reading “FDA Rejects Enzyvant’s Regenerative Therapy for Rare Immune Disorder”

Sage Therapeutics Depression Drug Fails in Phase 3, Shares Fall 60%

An experimental Sage Therapeutics drug intended to alleviate depression symptoms quickly failed to beat a placebo in a pivotal study, the company reported Thursday. Cambridge, MA-based Sage (NASDAQ: [[ticker:SAGE]]) has been testing its drug, SAGE-217, as a treatment for the symptoms of major depressive disorder. Unlike many depression drugs, which need weeks to take effect … Continue reading “Sage Therapeutics Depression Drug Fails in Phase 3, Shares Fall 60%”

As Alzheimer’s Conference Kicks Off, All Eyes Are on Biogen’s Drug

It’s the “era of neuroscience”; an industry “turning point”; and “an amazing time”—superlatives were flowing freely as the 12th annual Clinical Trials on Alzheimer’s Disease congress (CTAD) kicked off in rainy San Diego late Wednesday afternoon. The atmosphere felt thick with anticipation as attendees massed in the downtown hotel where the conference is taking place, … Continue reading “As Alzheimer’s Conference Kicks Off, All Eyes Are on Biogen’s Drug”

Avrobio’s Gabriel Cohn Joins Homology as Chief Medical Officer

Homology Medicines (NASDAQ: [[ticker:FIXX]]) has appointed Gabriel Cohn to serve as its chief medical officer. Cohn was most recently vice president of clinical development at Avrobio (NASDAQ: [[ticker:AVRO]]). His experience also includes positions at Shire, Baystate Medical Center, and the Tufts University School of Medicine. Homology, which is developing gene-editing medicines and gene therapies, raised … Continue reading “Avrobio’s Gabriel Cohn Joins Homology as Chief Medical Officer”

Civetta Therapeutics Unveils a Propeller Protein Plan, $53M in Funding

New biotech company Civetta Therapeutics has closed a $53 million Series A financing round to develop drugs that target “propeller proteins” as a way of treating cancers and other diseases. Propeller proteins are found throughout nature, and the circularly arranged “blades” of these proteins commonly serve as active sites to which molecules can bind. Bruce … Continue reading “Civetta Therapeutics Unveils a Propeller Protein Plan, $53M in Funding”

Digital Health Survivors & Innovative Incumbents Must Team Up for True Disruption

Editor’s note: This is Part 2 of a two-part post about innovation in digital health, co-authored by Rob Coppedge, CEO of Echo Health Ventures. Read Part 1 here. The Path Forward Between Echo Health Ventures and Blue Cross and Blue Shield of North Carolina, we work deeply on both sides of these partnerships and have perspective … Continue reading “Digital Health Survivors & Innovative Incumbents Must Team Up for True Disruption”

Repare Therapeutics Taps Clementia Pharma’s Steve Forte As Its New CFO

Repare Therapeutics said Tuesday it has appointed Steve Forte as executive vice president and chief financial officer. The company, which has offices in Cambridge, MA and Montreal is developing immune-oncology drugs by leveraging a concept known as synthetic lethality. In 2017 it emerged from stealth with $68 million in outside financing to advance its efforts; in September it … Continue reading “Repare Therapeutics Taps Clementia Pharma’s Steve Forte As Its New CFO”

Partner or Die: How to Succeed Even After the Digital Health Hype Quiets Down

Editor’s note: This is Part 1 of a two-part piece on innovation in digital health, co-authored by Bryony Winn, chief strategy and innovation officer at Blue Cross and Blue Shield of North Carolina. Read Part 2 here. More than 6,500 people descended on HLTH in Las Vegas in late October to “solve the most pressing … Continue reading “Partner or Die: How to Succeed Even After the Digital Health Hype Quiets Down”

Caraway Therapeutics Names Martin Williams as CEO

Cambridge, MA-based Caraway Therapeutics has named Martin Williams as its CEO. Williams joins the company, which is looking to treat neurodegenerative diseases by preserving neurons, from Yuma Therapeutics where he was the executive chairman. He will remain on Yuma’s board of directors. With more than 30 years of industry experience, Williams has held several leadership … Continue reading “Caraway Therapeutics Names Martin Williams as CEO”

IFM’s Glick Becomes Executive Chairman, Seidel Promoted to CEO

Gary Glick, co-founder and CEO of IFM Therapeutics, is moving on from his executive role to become executive chairman of the company’s board of directors. To take his place, Boston-based IFM promoted Martin Seidel, the company’s vice president of research and development, to CEO. He is also joining the company’s board. The personnel changes came … Continue reading “IFM’s Glick Becomes Executive Chairman, Seidel Promoted to CEO”

Agenus Chief Technology Officer Alex Duncan to Depart in January

Alex Duncan, chief technology officer of Agenus (NASDAQ: [[ticker:AGEN]]) for nearly four years, is leaving on Jan. 2, the company disclosed in a securities filing Friday. No reason was given for Duncan’s departure. Lexington, MA-based Agenus develops cancer drugs. Last year, Agenus struck a $150 million deal with Gilead Sciences (NASDAQ: [[ticker:GILD]]), which acquired global rights … Continue reading “Agenus Chief Technology Officer Alex Duncan to Depart in January”

IFM Lands $55.5M to Launch NewCo Quattro and Discovery Incubator

IFM Therapeutics has raised $55.5 million to launch its third drug subsidiary as well as an incubator, both of which are focused on developing new therapies for inflammatory diseases and cancers. Those endeavors will be led by a familiar face. Boston-based IFM has promoted its vice president of research and development, Martin Seidel, to become … Continue reading “IFM Lands $55.5M to Launch NewCo Quattro and Discovery Incubator”

La Jolla Pharma’s Share Price Halves On Trial End, CEO Departure

La Jolla Pharmaceutical is turning its focus to its lone FDA-approved product and another one in the pipeline after an experimental drug for a rare blood disease failed a late-stage study. On Monday, La Jolla Pharma (NASDAQ: [[ticker:LJPC]]) said it was halting a clinical trial testing LJPC-401, its drug candidate for iron overload in patients … Continue reading “La Jolla Pharma’s Share Price Halves On Trial End, CEO Departure”

Global Blood’s Sickle Cell Drug Wins FDA Approval Ahead of Schedule

The FDA on Monday approved a Global Blood Therapeutics treatment for sickle cell disease, three months before the agency’s decision was expected. Regulators evaluated the drug, voxelotor (Oxbryta), under a pathway that allows for a speedier review using less evidence than is typically required. This pathway is reserved for drugs that address an unmet medical … Continue reading “Global Blood’s Sickle Cell Drug Wins FDA Approval Ahead of Schedule”

Ferring and Blackstone Team Up to Fund Gene Therapy Firm With $570M

Ferring Pharmaceuticals and Blackstone Life Sciences have formed a new company to commercialize a bladder cancer gene therapy that is currently in late-stage clinical testing. The new company, FerGene, is a subsidiary of Swiss pharmaceutical company Ferring, which is investing $170 million in the new unit. Blackstone has committed $400 million. In addition to US … Continue reading “Ferring and Blackstone Team Up to Fund Gene Therapy Firm With $570M”

Axcella Taps Incyte’s Laurent Chardonnet as Chief Financial Officer

Axcella Health (NASDAQ: [[ticker:AXLA]]) announced it has hired Laurent Chardonnet as its chief financial officer. Chardonnet previously spent 15 years in a variety of finance-related roles at Incyte (NASDAQ: [[ticker:INCY]]), during which time it grew from a clinical-stage outfit to a $20 billion commercial-stage biopharma. Cambridge, MA-based Axcella, which was founded by Flagship Pioneering under … Continue reading “Axcella Taps Incyte’s Laurent Chardonnet as Chief Financial Officer”

Novartis to Acquire MedCo and FDA-Ready Cholesterol Drug for $9.7B

Novartis has agreed to acquire The Medicines Company for $9.7 billion, a cash deal that brings it a compound that is poised to become part of a new wave of cholesterol-lowering drugs. With the acquisition, Swiss pharma giant Novartis (NYSE: [[ticker:NVS]]) aims to compete with two other next-generation cholesterol drugs now being marketed by Amgen … Continue reading “Novartis to Acquire MedCo and FDA-Ready Cholesterol Drug for $9.7B”

Kymera Promotes Nello Mainolfi to President & CEO

Kymera Therapeutics’ new leader comes from within. Nello Mainolfi, the company’s co-founder, president, and chief scientific officer, has been promoted to president and CEO. Mainolfi’s experience includes positions at the Novartis Institutes for Biomedical Research, Atlas Venture, and Raze Therapeutics. He is succeeding former Kymera CEO Laurent Audoly, who announced in July that he would … Continue reading “Kymera Promotes Nello Mainolfi to President & CEO”

Bio Roundup: So Long Celgene, RNAi’s Arrival, Pharma in 2020 & More

Bristol-Myers Squibb this week completed its acquisition of Celgene, a $74 billion deal that creates a pharmaceutical juggernaut. And all it took was the largest asset sale ever required by federal antitrust regulators. As the Federal Trade Commission reviewed Bristol’s (NYSE: [[ticker:BMS]]) deal to acquire Celgene, anti-inflammatory drug apremilast (Otezla) emerged as a sticking point. … Continue reading “Bio Roundup: So Long Celgene, RNAi’s Arrival, Pharma in 2020 & More”

With Cell-By-Cell Take on Drug Discovery, Immunitas Debuts With $39M

A cancer tumor is a veritable patchwork of cells with a variety of genetic fingerprints. Immunitas Therapeutics is using single-cell genomics—an approach that studies the genetic activity of individual cells—to peer deeply into patient tumors and more precisely determine what is fueling the growth. With that knowledge, the company plans to develop new targets for … Continue reading “With Cell-By-Cell Take on Drug Discovery, Immunitas Debuts With $39M”

Former Shire Exec Robertson Joins Yumanity as Chief Medical Officer

Cambridge, MA-based Yumanity Therapeutics has named Brigitte Robertson to the role of chief medical officer. Most recently, she was the therapeutic area head of neuroscience global clinical development at Shire, which was then acquired by Takeda (NYSE: [[ticker:TAK]]). Robertson also held roles at Sunovion Pharmaceuticals in clinical development and experimental medicine, and GlaxoSmithKline (NYSE: [[ticker:GSK]]) … Continue reading “Former Shire Exec Robertson Joins Yumanity as Chief Medical Officer”

Relay Therapeutics Names Ben Wolf Chief Medical Officer

Relay Therapeutics has appointed Ben Wolf to serve as chief medical officer, the same position he held most recently at KSQ Therapeutics. His experience also includes positions at Blueprint Medicines (NASDAQ: [[ticker:BPMC]]), Merrimack Pharmaceuticals (NASDAQ: [[ticker:MACK]]), ImmunoGen, Amgen (NASDAQ: [[ticker:AMGN]]), and Genentech. Last year, Cambridge, MA-based Relay raised $400 million for its work developing cancer … Continue reading “Relay Therapeutics Names Ben Wolf Chief Medical Officer”

Alnylam Gets Quick FDA OK for Second RNAi Rare Disease Drug

[Updated 11/21/19, 10:05 a.m. See below.] An Alnylam Pharmaceuticals drug developed to treat a rare, inherited metabolic disorder won FDA approval on Wednesday, the second regulatory nod the company has received for a drug that turns off a gene to prevent it from causing disease. The FDA approved the Alynlam (NASDAQ: [[ticker:ALNY]]) drug givosiran (Givlaari) … Continue reading “Alnylam Gets Quick FDA OK for Second RNAi Rare Disease Drug”