Massachusetts

They Call It ‘Tranq’ — And It’s Making Street Drugs Even More Dangerous

Approaching a van that distributes supplies for safer drug use in Greenfield, Massachusetts, a man named Kyle noticed an alert about xylazine. “Xylazine?” he asked, sounding out the unfamiliar word. “Tell me more.” A street-outreach team from Tapestry Health Systems delivered what’s becoming a routine warning. Xylazine is an animal tranquilizer. It’s not approved for …

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GSK pays $100M to partner on a cancer drug with new twist on immunotherapy

GSK is partnering with Mersana Therapeutics on the development of an antibody-drug conjugate that is approaching Phase 1 testing. Unlike other ADCs that deliver a toxic drug payload, the Mersana drug is designed to activate the innate immune system to fight tumor cells.

Alnylam drug succeeds in heart study, setting stage for FDA filing and showdown with Pfizer

Alnylam Pharmaceuticals drug Onpattro, an FDA-approved treatment for nerve pain caused by hereditary transthyretin amyloidosis, now has Phase 3 data showing it can also help the much larger group of patients suffering heart problems from the rare protein disease. An FDA submission is planned, which would pit the Alnylam drug in competition with a blockbuster …

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Aiming to bring drug combos into earlier lines of cancer therapy, IDRx lands $122M

Taking inspiration from the success of drug cocktails that halt viral infections, startup IDRx aims to develop cancer drug combinations that stop tumor escape. The company, founded by serial entrepreneur Alexis Borisy, has set gastrointestinal stromal tumor (GIST) as its first disease target.

FDA hits pause on Beam Therapeutics’ off-the-shelf cell therapy for blood cancers

Beam Therapeutics’ application to begin human testing of a cell therapy made with base-editing technology is now under an FDA clinical hold. In other clinical hold news, the FDA lifted a hold on a Celyad Oncology cell therapy clinical trial that was halted in March.

U.S. government places $1.74B order for Moderna’s new Covid-19 vaccine boosters

The U.S. government has signed a supply agreement securing 66 million doses of Moderna’s Covid-19 vaccine booster shots, which are designed to address the omicron subvariants driving new cases of infection. The agreement follows a similar supply agreement reached with Pfizer and BioNTech.

Novartis turns to algorithms developer Anumana to apply AI to cardio conditions

Novartis is partnering with algorithms startup Anumana to develop a new artificial intelligence-based solution for cardiovascular diseases. The goal is to find ways to detect heart conditions years before symptoms develop in order to enable earlier intervention.

Amgen joins Kernal Bio’s $25M financing as the startup brings mRNA 2.0 to cancer

Kernal Biologics has technology that enables the delivery of messenger RNA therapies to a wider range of destinations in the body. The Series A financing comes as the startup works to advance its lead immuno-oncology program toward its first clinical test.

With new gene therapy data in hand, Sarepta talks with FDA about approval pathways

Sarepta Therapeutics has more clinical data showing the safety and efficacy of its experimental gene therapy for Duchenne muscular dystrophy. A pivotal Phase 3 test is already underway and could post data next year but the company is also talking with regulators about the possibility of a submission under the accelerated approval pathway.

New data for Intellia’s CRISPR therapy shows potential for one-time treatment of a rare disease

An Intellia Therapeutics therapy that uses CRISPR to perform in vivo genomic edits has clinical data showing that the therapeutic effects continue for a year in patients. The biotech now plans to speak with regulators about the design for a pivotal clinical trial testing this therapy, a potential one-time treatment for a rare inherited disease.

GSK gets pneumococcal vaccine contender with $2.1B Affinivax acquisition

Pfizer and Merck have each won FDA approvals in the past year for new pneumococcal vaccines, but GSK aims to top both of them with its $2.1 billion acquisition of Affinivax. That biotech’s Phase 3-ready vaccine candidate addresses more bacterial strains than any pneumococcal vaccines currently available.

New Flagship Pioneering startup expands the protein universe to fuel drug discovery

ProFound Therapeutics emerged from stealth with technology that it claims has discovered tens of thousands of previously undiscovered proteins—many of them promising new drug targets. The Flagship Pioneering-founded startup is backed by $75 million to support its drug R&D along with plans to partner with large pharmaceutical companies.

Innoviva’s Entasis acquisition brings a novel antibiotic that’s ready for the FDA

Entasis Therapeutics is being acquired by Innoviva in a deal that brings an FDA-ready antibiotic developed to address drug-resistant strains of a bacterium that infects critically ill, immunocompromised patients. Innoviva has been steadily building up its stake in Entasis, an AstraZeneca spinout, over the past two years.

CAR T without the toxicity: Kelonia gets $50M for a new take on cell therapy

Kelonia Therapeutics is developing cell therapies made by the in vivo engineering of a patient’s cells made possible by the targeted delivery of a genetic payload. The startup, newly out of stealth with $50M in funding, claims its approach would offer manufacturing and safety advantages over other cell therapy technologies.

FDA sounds caution on Foghorn cancer drug with a partial hold after patient death

The lead cancer drug candidate of Foghorn Therapeutics is now under a partial clinical hold following a patient death. According to the biotech, the patient may have experienced a side effect that is a known complication of leukemia drugs.

Enanta RSV drug fails a Phase 2 test, but studies continue in higher-risk patients

An antiviral that Enanta Pharmaceuticals is developing for respiratory syncytial virus infection failed to beat a placebo in its first Phase 2 test. But the biotech notes that these study participants weren’t very sick and may have recovered on their own, so it’s now pinning its hopes on ongoing studies in higher-risk groups, such as …

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Massachusetts reports first confirmed U.S. case of monkeypox this year

Massachusetts on Wednesday reported the first confirmed U.S. case of monkeypox this year in a man who had recently travelled to Canada. The case comes amid an ongoing outbreak of monkeypox cases in Europe occurring from an unclear source of transmission. “The Massachusetts Department of Public Health today confirmed a single case of monkeypox virus …

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Flagship merges two of its biotechs to form cellular microenvironment-focused Sonata

Sonata Therapeutics is developing drugs that reprogram cellular microenvironments to send signals intended to have curative effects. Cancer is the initial focus of the biotech, which Flagship Pioneering formed by combining two of its preclinical portfolio companies.

Claiming Akebia agreement breach, Otsuka seeks to end anemia drug pact

Otsuka Pharmaceutical is ending its alliance with Akebia Therapeutics, a move that comes less than two months after the FDA rejected their partnered anemia drug, vadadustat, and asked for another clinical trial. Otsuka alleges unspecified breaches of the agreement by Akebia, and ending the alliance early could spare it from spending hundreds of millions of …

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PepGen IPO nabs $108M for muscular dystrophy drug with potential edge over Sarepta med

PepGen’s technology improves the delivery of a therapy to more tissue types and the clinical-stage biotech plans to use its IPO cash to continue developing its lead program for Duchenne muscular dystrophy. Meanwhile, eye products giant Bausch + Lomb returned to the public markets as a standalone company. Both the PepGen and Bausch + Lomb …

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Biogen gets in tune with MedRhythms to develop new digital therapeutic for MS

Biogen struck a deal that could bring a digital therapeutic to its multiple sclerosis product portfolio. The drugmaker is partnering with startup MedRhythms in the development of a therapy that combines sensors, software, and music to improve walking problems in multiple sclerosis patients.

States Watching as Massachusetts Takes Aim at Hospital Building Boom and Costs

A Massachusetts health cost watchdog agency and a broad coalition including consumers, health systems, and insurers helped block the state’s largest — and most expensive — hospital system in April from expanding into the Boston suburbs. Advocates for more affordable care hope the decision by regulators to hold Mass General Brigham accountable for its high …

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Biogen pulls Alzheimer’s drug application in Europe as EMA calls data insufficient

Biogen withdrew an application seeking approval in Europe for Alzheimer’s disease drug Aduhelm, a move that follows a negative opinion on the therapy issued by a European Medicines Commission committee last December. The company was unable to persuade European regulators to change their minds about the drug.

Regenerative med biotech Satellite unveils tissue-based tech to restore organ function

Satellite Bio has emerged from stealth with technology for bioengineering tissue to restore organ function. The regenerative medicine startup, based on research from MIT and Boston University, is backed by $110 million in financing.

Cell therapy developer Be Bio hauls in $130M for pipeline in rare disease, cancer

Be Biopharma is developing cell therapies by engineering B cells to churn out therapeutic proteins with an initial focus on cancer and rare disease. The company’s Series B financing comes as competitors in the space also make progress with their engineered B cell therapies.

Microbiome biotech Kaleido Biosciences shutters amid cash crunch, drug setbacks

Kaleido Biosciences, formed by Flagship Pioneering as part of a wave of biotech companies developing microbiome therapies, has laid off its entire workforce and is winding down operations. The shutdown follows a strategic review that failed to turn up any deals to support its clinical-stage pipeline.

Final CMS decision limits coverage of Biogen’s Aduhelm to those in clinical trials

Biogen’s Alzheimer’s disease drug Aduhelm will only be covered for Medicare beneficiaries participating in a clinical trial. The final coverage determination will have the effect of limiting use of the drug, but the agency said its decision provides clarity on what drug companies need to show in order to secure Medicare coverage.

FDA places partial hold on Curis cancer drug after patient death in clinical trial

The FDA placed a partial hold on a Curis drug in development for treating leukemia after a patient treated in a clinical trial died. The biotech company said the patient experienced a complication that is a known risk of the small molecule drug.

Safety concerns sink Akebia drug for anemia from chronic kidney disease

The FDA rejected Akebia Therapeutics drug vadadustat as a treatment for anemia caused by chronic kidney disease. According to the biotech, the agency cited safety concerns and asked for another clinical trial to show that the pill’s benefits outweigh its risks.

Celsius Therapeutics heats up with $83M and an IBD drug on path to the clinic

Single-cell analysis biotech Celsius Therapeutics has raised $83 million to advance its pipeline of drugs for autoimmune disorders and cancer. The startup’s lead autoimmune program, a TREM1-blocking antibody for inflammatory bowel disease, is expected to begin human testing in early 2023.

GSK joins LifeMine’s $175M funding as partners revive fungi as a drug discovery frontier

LifeMine Therapeutics, a company that analyzes fungal genomes to find molecules that have potential as new medicines, has raised $175 million in financing. Among the investors is GlaxoSmithKline, which is teaming up with the biotech startup in a multi-target drug discovery alliance.

Apellis Pharma eyes a shot at getting first FDA approval in rare vision disorder

Apellis Pharmaceuticals has additional clinical data that show its drug having an effect on geographic atrophy, a rare vision-loss disorder with no FDA-approved treatment. The biotech plans to include the latest results in a new drug application being readied for submission in coming months.

Novartis turns to Voyager Therapeutics to get gene therapies into the brain

Novartis is paying Voyager Therapeutics $53 million up front to begin a partnership focused on the discovery of capsids for central nervous system disorder gene therapies. Depending on the progress of the research, Voyager could earn up to $1.5 billion in milestone payments.

Sherlock Biosciences secures $80M to bring molecular diagnostics to the home

Diagnostics developer Sherlock Biosciences has advanced its molecular diagnostic capabilities, which can now produce faster results on low-cost devices. The startup will use its Series B round of funding to expand the reach of its technology, potentially placing it directly in the hands of consumers around the world.

Flagship’s Vesalius launches with $75M to take AI into realm of common diseases

Artificial intelligence is a powerful tool for rare disease drug discovery. The founders of Vesalius Therapeutics say AI and machine learning can also be applied to common diseases, and the startup has launched with $75 million in committed financing.

Startup hC Bioscience emerges with $24M to join pursuit of transfer RNA therapies

New startup hC Bioscience is developing therapies based on transfer RNA, molecules that can be leveraged to address disease-causing proteins. The biotech joins several companies that are developing tRNA therapies in this emerging class of genetic medicines.

Agios strategy shift pays off with FDA approval of first drug for rare anemia

Agios Pharmaceuticals has its third FDA-approved product, and the first under its new rare disease strategy. The regulatory decision also marks the first approved treatment for pyruvate kinase deficiency, a rare disease that leads to chronic anemia.

KHN’s ‘What the Health?’: It’s Health Costs, Stupid (2022 Edition)

Can’t see the audio player? Click here to listen on Acast. You can also listen on Spotify, Apple Podcasts, Stitcher, Pocket Casts or wherever you listen to podcasts. The pandemic may be showing signs of winding down, but health care costs are not. In Massachusetts, a fight is shaping up over whether one of the …

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J&J partners with startup Remix, aiming to treat disease by “tuning” RNA

Several biotech companies are rising to the challenge of developing small molecules to drug RNA. Remix Therapeutics is joining the chase, but its different approach has drawn the interest of Johnson & Johnson, which has inked an R&D alliance spanning cancer and immunology.

What Does It Say About Your Neighborhood If the Supermarket Isn’t So Super?

Though I grew up in Roxbury, “the heart of Black culture in Boston,” I now live in Los Angeles, where I typically shop for groceries at Whole Foods Market or Trader Joe’s. Their produce is fresh, green, abundant. Organic options beckon as you walk in the door. So it gnawed at me, a Black woman, …

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Neuro-focused Arkuda raises $64M as dementia drug moves closer to the clinic

Eli Lilly is among the new investors joining the Series B round of Arkuda Therapeutics, a biotech developing a drug to treat a rare, inherited form of dementia. If the biotech’s approach works, it could also have applications in other neurodegenerative conditions such as Alzheimer’s and Parkinson’s.

Seizing “magic moment” for machine learning, startup Seismic unveils $101M

Machine learning is core to the drug research of Seismic Therapeutic, a new biotech startup developing novel drugs for immunology indications. CEO Jo Viney said the company’s initial focus is addressing dysregulation of adaptive immunity.

Intellia’s Rewrite Therapeutics buyout brings new precision to gene editing

Gene-editing medicines developer Intellia Therapeutics is paying $45 million up front to acquire Rewrite Therapeutics. The startup’s technology brings Intellia another tool for its genetic medicines toolbox, one that could enable even more precise edits.

Resistance to a Boston Hospital’s Expansion Centers on Rising Prices

A boisterous political battle over a proposed expansion by the largest and most expensive hospital system in Massachusetts is spotlighting questions about whether similar expansions by big health systems around the country drive up health care costs. Mass General Brigham, which owns 11 hospitals in the state, has proposed a $2.3 billion expansion including a …

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CMS proposes limited coverage of Biogen Alzheimer’s drug, only in clinical trials

The Center for Medicare and Medicaid Services proposes covering Biogen Alzheimer’s disease drug Aduhelm only for people enrolled in a clinical trial. In explaining this proposal, the agency cited the unclear benefit to patients as well as the documented safety risks associated with the drug, which can be best monitored within the context of a …

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Startup Avenge Bio nabs $45M for novel cell therapy approach to ovarian cancer

The field of biotechs pursuing new interleukin-2 cancer immunotherapies is filled with companies engineering different versions of that signaling protein. Avenge Bio CEO Mike Heffernan said his startup stands apart with a cell therapy that produces human IL-2 that could offer advantages compared to engineered versions of that protein.

Avrobio shifts gene therapy priorities as data surprise shelves lead program

Avrobio is stopping enrollment in a clinical trial for its Fabry disease program after new results showed variability in how the gene therapy was taken up by patients. No safety problems were reported, but the biotech said it now plans to focus its other gene therapy candidates for rare metabolic disorders.

FDA sets decision date for Amylyx Pharma ALS drug that improves neuron survival

The FDA has accepted Amylyx Pharmaceuticals’ submission for amyotrophic lateral sclerosis drug AMX0035 and it expects to issue a regulatory decision by late June. A lot will happen before then. Amylyx recently filed for an IPO and the company’s drug is slated for an advisory committee meeting ahead of an FDA decision.

Biogen projects confirmatory study for Alzheimer’s drug will take four years

Six months after Biogen was awarded regulatory approval for its Alzheimer’s disease drug Aduhelm, the company the company is laying out a timeline for the post-marketing study to confirm whether the drug works. The projected four-year study will enroll more than 1,300 patients and will include a placebo control arm.

Startup Mythic Therapeutics emerges with $103M to control fate of ADC cancer drugs

Antibody drug conjugates deliver a targeted strike to tumors, but the toxic payloads of these therapies can still reach healthy tissue. Mythic Therapeutics’ FateControl technology ensures that more of an ADC’s drug payload reaches the tumor, and CEO Alex Nichols is steering the startup out of stealth with $103 million and a lead program in lung …

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Eli Lilly steers into Foghorn’s gene traffic control drugs with $380M pact in cancer

Eli Lilly is building its cancer drug pipeline via a research alliance with Foghorn Therapeutic, a biotech whose technology discovers drugs addressing targets in a cellular system that regulate the way genes are turned on and off. In paying its new partner $380 million up front, Lilly is also placing yet another bet on targeted …

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After Phase 3 failure busts blockbuster bid, Deciphera restructures; staff slashed 35%

Deciphera Pharmaceuticals’ corporate restructuring will slash headcount by 35% and stop work on two drug programs. Going forward, the biotech said it will focus on developing drugs that have the potential to be the first or the best in their therapeutic classes.

Blueprint Medicines to buy cancer drug startup with potential edge over Takeda, J&J targeted therapies

Blueprint Medicines is paying $250 million up front to acquire Lengo Therapeutics, developer of targeted cancer therapies. The startup’s lead drug candidate is on track to begin human testing in non-small cell lung cancer, and Blueprint believes the small molecule has advantages over new targeted therapies from Johnson & Johnson and Takeda Pharmaceutical.

Generate Bio adds $370M for big push toward programmable protein medicines

Generate Biomedicines applies artificial intelligence and machine learning to protein analysis, which it uses to program its protein therapies for particular applications. The startup now has $370 million in financing to rapidly scale up operations, with a goal of reaching the clinic within two years.

Novo Nordisk reaches $3.3B deal to acquire RNAi partner Dicerna Pharma

The acquisition agreement comes exactly two years after the two companies began a wide-ranging alliance focused on developing RNA interference drug for metabolic disorders. The most advanced therapy from that alliance is on track to reach the clinic in 2022.

RA Capital reveals next play in protein degradation, this time outside the cell

The first wave of biotechs developing drugs that employ targeted protein degradation target disease-causing proteins inside the cell. Biotech startup Avilar Therapeutics, formed by RA Capital Management and led by CEO Dan Grau, is targeting proteins outside of the cell and it’s out of stealth backed by $60 million.

CRISPR-editing biotech Arbor Bio adds $215M for liver, CNS disease therapies

Arbor Biotechnologies raised new cash to keep up the pace in the chase for new genetic medicines. The MIT spinout applies artificial intelligence and machine learning to discover CRISPR enzymes applicable for gene-editing therapies; the Series B financing comes as programs in liver and central nervous system disorders move closer to human testing.

Flagship’s next genetic meds startup has $50M for transfer at a critical protein stop

Alltrna, a biotech startup founded by Flagship Pioneering, is developing a new type of medicines based on a form of RNA called transfer RNA, or tRNA. The company’s scientists say that this approach offers the potential to treat a wide range of diseases, both rare and common.

Biotech startup Marengo unveils $80M and a new way to get T cells to fight cancer

Apple Tree Partners’ latest cancer biotech company, Marengo Therapeutics, is using antibodies to selectively activate T cells to fight cancer. Led by former Merck KGaA executive Zhen Su, the startup is launching with $80 million in financing, which will support a drug pipeline that includes a lead program expected to reach the clinic in 2022.

Cell therapy biotech PlateletBio reels in $75M as it looks ahead to first clinical test

PlateletBio trails Sanofi among others in pursuit of a new therapy for a rare blood clotting disorder that has few treatments. But the biotech startup could stand apart from those drugs with its new class of cell therapy, which harnesses the properties of platelets.

Lonza Collaborates with Codiak to Acquire its Exosomes Manufacturing Facility in Lexington, Massachusetts (US)

Shots: Codiak to receive $65M of in-kind manufacturing services for its clinical-stage programs and hold its pipeline of therapeutic candidates along with exosome engineering and loading technologies Lonza will obtain global access and sub-licensable rights of Codiak’s high-throughput perfusion-based cGMP process for exosome manufacturing The companies established a center of excellence to develop exosome manufacturing …

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ALS drugmaker steps up with FDA filing, but yet another biotech stumbles in clinic

Amylyx Pharmaceuticals has submitted its amyotrophic lateral sclerosis (ALS) drug for FDA review based on positive Phase 2 data after discussions with the agency over the summer. Meanwhile, a Clene Nanomedicine ALS drug failed its Phase 2 study, but the biotech points to encouraging results in a subgroup of patients.

3 States Limit Nursing Home Profits in Bid to Improve Care

Nursing homes receive billions of taxpayers’ dollars every year to care for chronically ill frail elders, but until now, there was no guarantee that’s how the money would be spent. Massachusetts, New Jersey and New York are taking unprecedented steps to ensure they get what they pay for, after the devastating impact of covid-19 exposed …

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Xilio, Ventyx IPOs raise $269M to fuel plans to compete with big pharma drugs

Cancer drug developer Xilio Therapeutics and Ventyx Biosciences, a company with drug candidates in cancer and autoimmune diseases, are the latest life science companies to go public. They’ll use proceeds from their respective IPOs to continue clinical development of drugs being positioned as competitors to drugs from big pharmaceutical companies.

FDA refusal of Stealth Bio drug shows challenges of ultra-rare disease studies

The FDA refused to review Stealth BioTherapeutics’ Barth syndrome drug, telling the company results in a study of just eight patients are insufficient to support its submission. The impasse highlights the challenges of testing drugs for ultra-rare diseases. Barth is so rare that Stealth is unsure it can recruit patients to run a new study.

Entasis antibiotic bests last-resort drug in Phase 3 trial; FDA filing planned for 2022

Entasis Therapeutics, the former anti-infectives unit of AstraZeneca, has positive Phase 3 data for its lead program, an antibiotic that could address drug-resistant strains of Acinetobacter baumannii that contribute to hospital-acquired infections. The biotech plans to seek FDA approval in 2022.

Biogen ALS drug fails main goal of key test, but company points to other results

Biogen amyotrophic lateral sclerosis drug, tofersen, failed a Phase 3 clinical trial, but the drug showed improvement according to secondary and exploratory goals of the study. Based on those results, the company said it is talking with regulators to find a path forward for the drug.

Resilience turns to Harvard for biologics manufacturing tech, inks 5-year R&D pact

Biomanufacturing technologies company Resilience is partnering with Harvard in a deal that could see innovations from the university’s labs commercialized under new businesses. Resilience has committed $30 million in funding to support Harvard biomanufacturing research.

Atlas Venture-incubated Rectify lands $100M to transport drug hunt to new grounds

Rectify Pharmaceuticals is taking an approach to disease that led to a successful franchise of Vertex Pharmaceutical lung drugs and expanding it to more diseases in more organs in the body. The startup has launched with $100 million in financing.

Pyxis IPO leads way as four life science firms kick off 4Q, raising nearly $500M

Pyxis Oncology’s IPO raised $168 million as the biotech steers toward the clinic with drug candidates and technology licensed from Pfizer. Theseus Pharma, IsoPlexis, and Cognition Therapeutics also went public; combined the four companies raised nearly half a billion dollars.

Allied with Amgen and backed by $500M, Neumora brings data science to brain drugs

Neumora Therapeutics unveiled $500 million in capital and a research partnership with Amgen. The startup analyzes neurological data to inform its drug research and development; President and Chief Operating Officer Lori Lyons-Williams said researching psychiatric drugs and neurodegenerative drugs within the same company allows brain insights from one area to inform the other.

Voyager Therapeutics’ strategy shift to gene therapy tech leads to a Pfizer alliance

The gene therapy pipeline of Voyager Therapeutics has hit several setbacks in the past year, but a strategy shift to its capsid technology is now showing promise. Pfizer has signed on as partner in a wager that Voyager’s tech holds the key to delivery of gene therapies for cardiovascular and neurological disorders.

Vedanta Bio guts out microbiome trial win to keep up with field, get BARDA cash

Vedanta Biosciences is developing its microbiome therapy with support from the Biomedical Advanced Research and Development Authority. Under terms of that contract, the positive Phase 2 results in preventing recurrent C. diff infection trigger a $23.8 million payment.

Exo Therapeutics adds $78M as cancer, inflammation drugs march toward clinic

Biotech startup Exo Therapeutics aims to overcome the challenges of drugging proteins by targeting exosites, locations that modulate enzyme activity. The company has four small molecule drug candidates for cancer and inflammation, and the company plans to use the $78 million in new financing to advance them toward human testing.

Anji Pharma adds $70M for clinical trials, expansion of ‘hub and spoke’ biz model

Cross-border biotech Anji Pharma closed $70 million in Series B funding to advance a drug pipeline that includes metabolic and cancer drugs. The biotech’s business model of finding promising drugs and forming subsidiaries to develop them is similar to approaches taken by Roivant Sciences and BridgeBio Pharma, among others.

Biogen and Samsung land FDA approval for biosimilar to blockbuster Roche drug

The FDA has approved the first biosimilar that references the blockbuster Roche drug Lucentis. The Biogen and Samsung BioLogics joint venture that developed the biologic product have approval to treat three eye conditions that lead to vision loss.

Repligen sees $150M Avitide acquisition as key to reaching gene therapy goals

Bioprocessing technologies company Repligen is acquiring Avitide, and its biologics purifying technology, in a $150 million cash and stock deal. Repligen said Avitide will help it meet growing demand for gene therapy solutions.

FDA warns Kaleido Bio to test its Covid-19 candidate as a drug, not a medical food

The FDA issued a warning letter to Kaleido Biosciences alleging the company tested its experimental microbiome product in Covid-19 patients without submitting a formal clinical trial application. Kaleido claims the filing was not required because the product is a medical food, but the regulator doesn’t buy that argument.

Florida Spine Surgeon and Device Company Owner Charged in Kickback Scheme

A Florida orthopedic surgeon and designer of costly spinal surgery implants was arrested Tuesday and charged with paying millions of dollars in kickbacks and bribes to surgeons who agreed to use his company’s devices. Dr. Kingsley R. Chin, 57, of Fort Lauderdale, Florida, is the founder, chief executive officer and owner of SpineFrontier, a device …

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Blood disorder biotech Disc Medicine gets $90M to bring two drugs into Phase 2

A Roche drug that failed as a treatment for neurological disorders is now the lead program for Disc Medicines. CEO John Quisel said Roche’s clinical data showed the small molecule’s promise addressing a rare blood disorder and now the biotech startup has $90 million to advance that drug and another one into Phase 2 testing.

A biotech startup’s hunt for elusive disease targets scores $60M for drug R&D

Atavistik Bio is one of several companies discovering and developing drugs that work by allostery, binding to less obvious sites of a target protein. Acting CEO John Josey said the startup aims to stand apart with its focus on understanding metabolic interactions, a path less trodden by others in allosteric drug discovery.

Pfizer makes big move in blood cancers with $2.3B Trillium acquisition

Though Gilead Sciences leads the group of companies developing immunotherapies that target the cancer protein CD47, Pfizer contends that molecules it is getting through the Trillium Therapeutics acquisition have safety and efficacy advantages that could make them the best in this emerging drug class.

Sesen Bio says rejected bladder cancer drug may need new clinical trial

For much of this summer, Sesen Bio said all of the FDA’s communications pointed toward regulatory approval for the biotech’s bladder cancer drug. Though Sesen characterized the regulator’s rejection of the fusion protein as surprising, CEO Tom Cannell also revealed an earlier sign that another clinical trial may be necessary.

Eli Lilly joins $30M Series A financing for startup bringing AI analysis to endoscopy

The artificial intelligence-based technology of Iterative Scopes brings computer vision analysis to endoscopic images. The startup’s technology was initially developed to assist gastroenterologists in finding pre-cancerous polyps but CEO and founder Jonathan Ng said it’s also finding additional use helping pharmaceutical companies identify patients for clinical trials.

Biogen and Eisai unveil details for five-year, real-world Aduhelm clinical trial

Patients taking new Alzheimer’s disease drug Aduhelm will be evaluated in a real-world study lasting up to five years. Biogen announced plans for the observational study, which is one of three post-marketing clinical programs planned to generate more data about the drug

Ring Therapeutics rounds up $117M to find better viral vectors for gene therapy

The engineered viruses used to deliver gene therapies can spark complications and they can’t be re-dosed. Ring Therapeutics says viruses that evolved with humans can be better viral vectors and the startup has raised $117 million in new financing to continue its research.

Seres Therapeutics microbiome drug flunks Phase 2 test in ulcerative colitis

Seres Therapeutics’ ambitions to treat ulcerative colitis with a microbiome drug were dealt a setback as its experimental therapy failed in Phase 2 testing. The company plans a “rigorous scientific analysis” to assess what happened and to determine the next steps for the program.

Amylyx Pharmaceuticals raises $135M as ALS drug heads to pivotal clinical trial

An amyotrophic lateral sclerosis drug from Amylyx Pharmaceuticals is being prepared for Phase 3 clinical testing on track to begin later this quarter. To support that research, the biotech has raised $135 million in financing.

PAQ Therapeutics scores $30M to unleash “Pac Man” drugs on neuro disease

Startup PAQ Therapeutics is developing drugs work like Pac Man, gobbling up components of a cell associated with disease. The biotech has closed $30 million in financing to continue its research, with a neurodegenerative disorder as its lead disease target.

Biogen reaches across globe and pays $125M to land its next new MS drug

By paying $125 million up front for rights to multiple sclerosis drug candidate orelabrutinib, Biogen secures a place among those companies trying to treat the disease by targeting a particular enzyme highly expressed in the central nervous system. But Biogen still has some catching up to do.

Sigilon hemophilia A cell therapy trial placed on hold after patient complication

A hemophilia A patient in a Sigilon Therapeutics clinical trial developed a well-known complication to treatments for the blood disorder. The potential problem for Sigilon is that its cell therapy is designed to avoid triggering such immune responses.

15 States Reach a Deal With Purdue Pharma Over Opioids

The states, including Massachusetts and New York, agreed to drop opposition to the bankruptcy organization plan of the company, the maker of OxyContin.

As Covid Vaccinations Slow, Parts of the US Remain Far Behind 70% Goal

July Fourth was not the celebration President Joe Biden had hoped for, as far as protecting more Americans with a coronavirus vaccine. The nation fell just short of the White House’s goal to give at least a first dose to 70% of adults by Independence Day. By that day, 67% of adult Americans had gotten …

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CRO Parexel changes private equity hands again, this time for $8.5B

Four years after a private equity acquisition took Parexel private, the contract research organization is being acquired by two private equity firms for $8.5 billion. It’s the latest in a series of acquisitions to hit the CRO sector this year.

Apellis taps Beam’s base-editing tech to broaden autoimmune disease scope

Fresh off its first FDA approval, Apellis Pharmaceuticals is teaming up with Beam Therapeutics, a partnership that will use Beam’s base-editing technology to develop new therapies for complement system disorders. Apellis has committed to pay Beam $75 million to kick off the alliance.

Intellia’s early CRISPR trial data validate a drug pipeline and the gene-editing field

Intellia Therapeutics has early clinical trial data showing that its CRISPR-based therapy for a rare disease can edit genes inside the body safely and effectively. The trial is ongoing, but the preliminary results suggest that Intellia’s approach could offer a cure.

Lyell, Verve IPOs raise a combined $692M as both biotechs steer toward clinical trials

Lyell Immunopharma and Verve Therapeutics are still preclinical, but they’re bring novel genetic approaches to the treatment of cancer and cardiovascular disease. Now the two companies can claim two of the biggest biotech IPOs of 2021 so far.

Northpond, 5AM Ventures lead $45M funding for CAMP4’s trek to new RNA therapies

CAMP4 Therapeutics is developing a new kind of RNA therapy that treats disease by upregulating gene expression. The startup has raised $45 million as CEO Josh Mandel-Brehm steers toward clinical trials expected to begin next year in liver and brain diseases.

While Sage Therapeutics sees a window for depression drug, market is skeptical

A Sage Therapeutics depression drug that failed a pivotal study in 2019 has positive preliminary results from a new Phase 3 clinical trial, but the data also showed the therapy’s effects waned over time. Questions about zuranolone’s durability are relevant to Biogen, which committed $1.5 billion to Sage in large part due to the drug’s …

While Sage Therapeutics sees a window for depression drug, market is skeptical Read More »

Pioneering a new realm of biology, startup Senda Bio expands financing to $98M

Senda Biosciences is developing drugs based on an understanding of intersystems biology—the way that humans interact with bacteria and plants. The startup, founded by Flagship Pioneering, has added $55 million to advance its three lead programs to clinical testing next year.

AI-drug discovery biotech Valo Health is going public via a $2.8B SPAC merger

Valo Health is going public in a SPAC merger. Less than nine months removed from its formal launch, Valo is now revealing more about its artificial intelligence platform technology, which it plans to use to make the firm the first “digitally native” pharmaceutical company.

MorphoSys to buy Constellation Pharma in a $1.7B bet on epigenetic cancer drugs

MorphoSys is acquiring Constellation Pharmaceuticals and its late-stage myelofibrosis drug in a deal that values the epigenetics biotech at $1.7 billion. Germany-based MorphoSys will finance the acquisition with cash from a separate $2 billion deal with drug royalty acquirer Royalty Pharma.

Moderna’s Covid vaccine shows efficacy in younger teens; regulatory filing are next

Moderna released preliminary clinical data showing its Covid-19 vaccine produced efficacy in adolescents that was comparable to what was seen in adults. The company plans to submit applications to regulators in early June seeking expansion of emergency authorization of the vaccine to include this younger age group.

Biogen teams up with Ginkgo Bioworks in gene therapy manufacturing pact

Biogen is reaching across the Charles River, striking up a gene therapy manufacturing alliance with synthetic biology firm Ginkgo Bioworks. Ginkgo will use its technology to accelerate and make more efficient the process of manufacturing the AAV viral vectors used in gene therapies.

Bayer’s BlueRock Therapeutics widens cell therapy scope to the eye under new pact

BlueRock Therapeutics, a Bayer subsidiary, is teaming up with Opsis Therapeutics and Fujifilm Cellular Dynamics in an R&D pact focused on developing stem cell therapies for eye diseases. BlueRock is paying its new partners $30 million up front to kick off the alliance.

Bristol Myers writes $200M check for rights to Agenus checkpoint inhibitor

Bristol Myers Squibb already has a cancer program targeting the TIGIT protein, but in acquiring rights to Agenus’s drug, the pharma giant adds a bispecific antibody that preclinical research suggests has the potential to be the best in this drug class. BMS agreed to pay $200 million up front for global rights.

With FDA approval, Apellis can challenge AstraZeneca in rare blood disorder

The FDA approved Apellis Pharmaceuticals drug pegcetacoplan (Empaveli) as a treatment for a rare blood disorder that is currently treated with medicines sold by Alexion Pharmaceuticals, a biotech being acquired by AstraZeneca. But Apellis’s drug beat Alexion drug Soliris in a head-to-head test, and the smaller company aims to steal market share from its larger …

With FDA approval, Apellis can challenge AstraZeneca in rare blood disorder Read More »

Biogen adds to neuro drug pipeline with acquisition of stroke drug from TMS

Biogen paid $18 million to acquire a drug that TMS Co. of Japan tested in patients with acute ischemic stroke. The acquisition follows the report of preliminary mid-stage data showing the drug led to no cases of a potentially fatal complication associated with currently available stroke therapies.

‘I Just Feel Like Myself’: A Nonbinary Child In Their Own Words

It’s 7:30 a.m. on a school day. Two parents are racing to get their three young children dressed, fed, packed for the day, into coats and out the door when 6-year-old Hallel runs downstairs, crying. This story is part of a partnership that includes WBUR, NPR and KHN. It can be republished for free. Ari, …

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Andreessen Horowitz leads $100M Series A round in Dyno and its gene therapy tech

Dyno Therapeutics’ technology for designing viral vectors that deliver gene therapies has led to partnerships with Novartis, Sarepta Therapeutics, and Roche. With the Series A financing, the startup plans to expand its technology to address more tissue types, and potentially add more partners.

Neuroelectrics gets $17M for new take on electrical therapy for epilepsy, depression

Neuroelectrics is planning a pivotal test of its wearable medical device, which delivers brain electrical stimulation to treat epilepsy. The startup’s Series A round of funding was led by Morningside Ventures.

Gene therapy funding frenzy reaches Affinia as it reels in $110M in new capital

Affinia Therapeutics, which has technology that could deliver gene therapies to more tissue types in the body, raised the Series B financing as it looks ahead to clinical testing. The biotech is the latest company to close a substantial round of funding for gene therapy technology.

Biotech IPO wave rolls on as Werewolf, Vaccitech raise $230M for clinical trials

Vaccitech and Werewolf Therapeutics joined the public markets, raising a combined $230 million. The vaccine developer and the cancer immunotherapy biotech will apply the IPO proceeds toward clinical development of their respective pipelines.

No more tears: Aldeyra’s eye drug data support its allergy treatment alternative

Seasonal allergy sufferers have a plethora of choices for treating the itching and redness affecting their eyes. An experimental Aldeyra Therapeutics drug that takes a new approach to blocking inflammation triggered by allergens has met the goals of a pivotal study and the biotech now plans to discuss the data with the FDA.

Pandemic Imperiled Non-English Speakers More Than Others

In March 2020, just weeks into the covid-19 pandemic, the incident command center at Brigham and Women’s Hospital in Boston was scrambling to understand this deadly new disease. It appeared to be killing more Black and brown patients than whites. For Latinos, there was an additional warning sign: language. This story is part of a …

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Alnylam FDA submission aims to expand biotech’s franchise of gene-silencing drugs

Alnylam Pharmaceuticals has filed for FDA approval of vutrisiran, which could become the biotech’s fourth drug that works by RNA interference. Meanwhile the company is responding to a federal subpoena seeking documents related to the company’s marketing and promotion of its first RNAi drug, Onpattro.

Vertex Pharma inks another R&D alliance to expand its gene-editing ambitions

Vertex Pharmaceuticals is collaborating with Obsidian Therapeutics to develop up to five new gene editing therapies. Obsidian’s technology precisely controls protein expression, a capability the partners plan to apply to several undisclosed serious diseases.

Regulatory progress for two biotechs stalls as FDA requests more information

The FDA placed a clinical hold on a KalVista Pharmaceuticals drug being readied for mid-stage studies in a rare, inherited disorder. Meanwhile, the FDA has some questions about the historical data Y-mAbs used as the control in a clinical trial for its antibody cancer drug.

Adagio adds $336M for pivotal tests of drug with potential to beat Covid variants

Antibody therapies are seen as one way to overcome the variants that continue to fuel the pandemic. With its Series C financing, Adagio Therapeutics, led by CEO Tillman Gerngross, will proceed with a pivotal clinical trial of an antibody that has neutralized SARS-CoV-2 variants in lab and animal tests.

Relay Therapeutics pays $85M for startup with a new AI tech for drug discovery

Relay Therapeutics has its own technology that applies artificial intelligence and machine learning techniques to drug discovery. Its $85 million acquisition of ZebiAI gives it another technology that applies ML to DNA-encoded libraries and predicts the small molecules with the potential to drug proteins.

Thermo Fisher’s $21B deal for PPD gives it missing piece in drug development cycle

Thermo Fisher Scientific sells laboratory equipment used in drug research and it also offers manufacturing services for experimental and commercialized drugs. By acquiring PPD, Thermo Fisher says it will gain capabilities in the growing global market of clinical trial services.

Amylyx Pharma’s ALS drug needs a Phase 3 study to support FDA submission

Amylyx’s ALS drug met the goals of a mid-stage study, paving the way for regulatory submissions in Europe and Canada. But the FDA is requiring data from a larger Phase 3 clinical trial and that study is slated to begin later this year.

Biotech startup Repertoire adds $189M to expand array of immune medicines

With two cancer programs already making progress, Repertoire Immune Medicines will use the Series B financing to expand its immune synapse research to autoimmune disorders and infectious disease. CEO John Cox said his startup’s approach could advance immune medicines beyond the scope of currently available therapies.

Ventus Therapeutics tacks on $100M to take on ‘undruggable’ disease targets

RA Capital led Ventus Therapeutics’ Series B financing, which the startup will use to develop its pipeline of medicines for “undruggable” disease targets. CEO Marcelo Bigal said the cash also gives Ventus the flexibility to consider an IPO.

Hologic makes a multiplex molecular testing move with $795M Mobidiag acquisition

Mobidiag offers multiplex testing, which is the ability to test for multiple pathogens from a single test sample. Hologic said the acquisition will enable the company to enter the acute care test market, which is projected to grow in a post-pandemic world.

Need Amid Plenty: Richest US Counties Are Overwhelmed by Surge in Child Hunger

Alexandra Sierra carried boxes of food to her kitchen counter, where her 7-year-old daughter, Rachell, stirred a pitcher of lemonade. This story also ran on USA Today and GateHouse Media. It can be republished for free. “Oh, my God, it smells so good!” Sierra, 39, said of the bounty she’d just picked up at a …

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Aveo Oncology’s kidney cancer drug gets FDA nod, 8 years after stinging rejection

In two pivotal studies years apart, Aveo Oncology’s drug tivozanib did not help kidney cancer patients live longer overall compared to the standard of care. The FDA approved still approved it, the latest development in a long saga that includes several regulatory stumbles.

Neurocrine nixes Parkinson’s gene therapy from Voyager Theraeutics alliance

The Neurocrine decision follows a clinical hold placed on the Parkinson’s program in December. It’s Voyager’s second loss of an R&D partner in the past year; AbbVie previously ended an alliance aiming to develop gene therapies for Parkinson’s and Alzheimer’s.

Estados permiten contratar profesionales de salud extranjeros por la pandemia

Cuando los hospitales de todo el país luchan contra la nueva ola de la pandemia, no son camas ni ventiladores lo que escasean. Son las personas que cuidan de los enfermos. Pero existe mano de obra altamente calificada de médicos, enfermeras y otros trabajadores de salud, con formación en el extranjero, que no se aprovecha …

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Evictions Damage Public Health. The CDC Aims to Curb Them ― For Now.

In August, Robert Pettigrew was working a series of odd jobs. While washing the windows of a cellphone store he saw a sign, one that he believes the “good Lord” placed there for him. “Facing eviction?” the sign read. “You could be eligible for up to $3,000 in rent assistance. Apply today.” It seemed a …

Evictions Damage Public Health. The CDC Aims to Curb Them ― For Now. Read More »

Moderna’s Covid-19 vaccine shows antibody, T-cell responses a month after second dose in interim Phase I data

The company published interim data from the Phase I study of mRNA-1273 in 40 older and elderly adults, showing responses comparable to those seen in younger adults. The vaccine is being tested in a 30,000-participant Phase III trial.

Foundation Medicine to launch liquid biopsy companion diagnostic following FDA approval

The company announced Wednesday the FDA approval of its liquid biopsy test, the second to win an agency nod in less than a month, saying it would launch the product on Friday. The FDA acquired the first ever liquid biopsy companion diagnostic, Guardant Health’s Guardant360 CDx, on Aug. 10.

Another COVID Mystery: Patients Survive Ventilator, But Linger in a Coma

Leslie Cutitta said yes, twice, when clinicians from Massachusetts General Hospital in Boston called asking whether she wanted them to take — and then continue — extreme measures to keep her husband, Frank Cutitta, alive. The first conversation, in late March, was about whether to let Frank go or to try some experimental drugs and …

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Study uses genomic data to trace Covid-19 ‘superspreader’ event linked to February Biogen exec meeting

The study, by researchers at Harvard, MIT, The Broad and other institutions, used genomic analysis of cases linked to the Biogen meeting, linking much of the community transmission in the Boston area to a European genetic variant of SARS-CoV-2 that first appeared at the meeting.

J&J to buy Momenta Pharmaceuticals for $6.5B

The drugmaker emphasized the full global rights it would acquire to Momenta’s lead asset, nipocalimab, which it is developing for several autoantibody-driven diseases and that in certain indications could have peak sales of more than $1 billion.

Contact Tracers in Massachusetts Might Order Milk or Help With Rent. Here’s Why.

It’s a familiar moment. The kids want their cereal and the coffee’s brewing, but you’re out of milk. No problem, you think — the corner store is just a couple of minutes away. But if you have COVID-19 or have been exposed to the coronavirus, you’re supposed to stay put. Even that quick errand could …

Contact Tracers in Massachusetts Might Order Milk or Help With Rent. Here’s Why. Read More »

Moderna secures more than $1.5B federal government deal to provide Covid-19 vaccine

The agreement, which includes 100 million doses of mRNA-1273 and an option to acquire 400 million more, brings the total amount of money the biotech company has received from the federal government for its vaccine efforts to nearly $2.5 billion.

Biogen to get speedy FDA review for Alzheimer’s drug despite not using voucher to request it

Biogen and Eisai said the FDA had accepted and given priority review to their application for aducanumab. The drug will undergo an advisory committee meeting at an undisclosed date. Biogen’s stock rose 10%, but analysts had mixed opinions.

Alexion drops kidney disease program for drug that was part of $930M Achillion buyout last year

The company said in its 2Q earnings that it would halt development of ALXN2040 for C3 glomerulopathy, or C3G, citing the drug’s lack of potency. However, it may develop a second, more potent drug from the Achillion acquisition, ALXN2050, in the same disease.

BMS, bluebird refile FDA approval application for CAR-T therapy in myeloma

The companies’ resubmission of their application Wednesday seeking approval for idecabtagene vicleucel was in line with the timeline they provided in May, when the FDA sent a refuse-to-file letter in response to their initial submission.

Thrive raises $257M Series B round for registration study of blood test for early cancer detection

The study design is still being finalized, and the company is thus not yet divulging too many details. However, CEO David Daly said it would be significantly larger than the previous DETECT-A, multi-center and would include both men and women.

Moderna snags another $472M from BARDA, launches Phase III Covid-19 vaccine trial

The company had received $483 million from BARDA in April to fund the development of the vaccine, mRNA-1273, through Phase III, but that amount was based on a smaller anticipated number of participants than the 30,000 it now plans to enroll into the trial, named COVE.

Biogen to launch Phase IV study of spinal muscular atrophy drug in post-gene therapy patients

The company plans to enroll 60 patients who have received its drug, Spinraza, following suboptimal response to Novartis’ Zolgensma, which was approved last year. The trial, RESPOND, is anticipated to start enrolling in the first quarter of 2021, pending regulatory approval.

Moderna publishes Phase 1 Covid-19 vaccine data showing robust immune responses

Evaluation of durability is still under evaluation, but the responses to mRNA-1273 show neutralizing antibody titers multiple times higher than those of recovered patients. Additionally, T-cell responses suggest potentially lower risk of enhanced respiratory disease.

Roche pays Blueprint $675M upfront for ex-U.S. development, commercialization rights to cancer drug

Under the deal, Blueprint will additionally be eligible for more than $900M in milestone payments plus royalties for the RET inhibitor pralsetinib. The deal includes exclusive rights for Roche in ex-U.S. markets other than China and shared rights in the U.S.

Biogen, Eisai complete submission of controversial Alzheimer’s drug. The ball is in FDA’s court now.

The FDA has 60 days to decide whether or not to accept the application. An analyst wrote that the agency accepting it with a priority or standard review may signal whether the agency is seriously considering approval or has continued reservations about the amyloid beta-targeting drug’s clinical benefit.