daniel-gordon/biopharma-dive huggingface dataset:
Rapports latest haul adds to an influx of funding over the past couple years for neuroscience-focused startups
Interest tax depreciation and amortization Interest expense net amounts can vary substantially from period to period due to changes in cash and debt balances and interest rates driven by market conditions outside of the Companys operations
Intellia conducted an earlier study of the therapy at sites in Europe the UK and New Zealand. Both treatments work in vivo meaning the gene editing caused by the therapies happens inside the body rather than in cells edited in a laboratory or ex vivo The FDA has so far been cautious in permitting testing of in vivo treatments to move ahead quickly
If approved Ascendis expect s its first European country launch in early 2024
With latest fundraise Rome Therapeutics works to drug the ʻdark genomeʼ Led by former Nimbus executive Rosana Kapeller the startup has now raised nearly 200 million in its pursuit of new autoimmune disease and cancer therapies
Results released this year showed that among 17 evaluable sickle cell patients 16 were free of pain crises for at least one year after receiving Vertex and CRISPR Therapeutics therapy
The Novo foundation hopes to usher more experimental projects into human trials
Mean vector genome copies per nucleus reached 387
Sanofis upfront payment of 500 million is modest compared to Mercks decision to pay 11 billion to acquire Prometheus but that company benefited from already having positive midstage data wrote Evercore ISI analyst Umer Raffat in a client note
FDA expands use of Neurocrine drug to Huntingtonʼs patients Analysts expect the approval to intensify the market competition between Neurocrineʼs Ingrezza and a similar drug from Teva
TSHA102 has received Orphan Drug and Rare Pediatric Disease designations from the FDA and has been granted Orphan Drug designation from the European Commission
In addition to private equity investments OrbiMed also offers companies nondilutive capital via its royalty and credit programs
Fulcrum shares surged about 50 in premarket trading Tuesday
VYONDYS 53 has met the full statutory standards for safety and effectiveness and as such is not considered investigational or experimental
Embark got its start at the Novo Nordisk Foundation Center for Basic Metabolic Research where research conducted by Zach GerhartHines Jakob Bondo Hansen and Thue Schwartz gained seed backing from Novo Holdings and a collaboration with Novo Nordisk itself
A brainfocused biotech raises 225M to develop drugs for schizophrenia Parkinsonʼs MapLight Therapeuticsʼ Series C round saw participation from new investors Novo Holdings 5AM Ventures and Cowen Healthcare Investments
Xphozah joins a number of existing treatments for hyperphosphatemia which is a common occurrence in people with kidney disease
The latter has become the focus of a pharma industry arms race boosted by the success of diabetes and weight loss drugs like Ozempic and Wegovy
Beam expects to complete the layoffs by the end of the year and said the related savings will allow it to fund operations into 2026. The company held 11 billion in cash and marketable securities as of the end of June a sum boosted by a lucrative deal with Pfizer last year
Biogen says growth is coming but wonʼt specify when the CEO said the company has the elements to think about a return to top-line growth. Yet that goal hinges on the successful launch of multiple new products
Gene therapy developer UniQure is laying off 114 employees and halting more than half of its research and technology projects the company said Thursday . The Dutch biotechnology company said the costcutting measures would save the company 180 million over the next three years extending its cash runway into 2027
Epigenic Therapeutics attracted investments from Qiming Venture Partners OrbiMed and existing investor Morningside Venture Capital in its latest funding round
3andMe has cemented its place as a goto provider of athome genetic testing over the past two decades
About Taysha Gene Therapies Taysha Gene Therapies Nasdaq TSHA is on a mission to eradicate monogenic CNS disease
The Biologics License Application BLA for SRP9001 is currently under priority review by the FDA with a regulatory action date of May 29 2023
hATTR amyloidosis aects approximately 50000 people worldwide while wtATTR amyloidosis is estimated to impact 200000  300000 people worldwide
The most common symptoms of IRRs with ONPATTRO were flushing back pain nausea abdominal pain dyspnea and headache
If a dose of VOXZOGO is missed it can be given within 12 hours from the missed dose
In March 2020 the Company completed its sale of the PRV and received proceeds of 1081 million net of commission which was recorded as a gain from sale of the PRV as it did not have a carrying value at the time of the sale
The most common side effects of VOXZOGO include injection site reactions including redness itching swelling bruising rash hives and injection site pain high levels of blood alkaline phosphatase shown in blood tests vomiting joint pain decreased blood pressure and stomachache
Nimbus hopes to prove the drug is useful as a monotherapy meaning it doesnt need to be taken in combination with other medicines to have an effect
Adlai Nortyes stock offering which the company initially set plans for in late July follows positive signs for what has otherwise been a moribund IPO market this year
We use our platform  to think about subpopulations and biomarkers but its the program that were excited about. We are building a platform in service of our pipeline.
A new biotechnology startup is getting help from an unusual partner in its attempt to develop rare disease drugs that could also treat more common conditions. Actio Biosciences  which launched Tuesday with 55 million in venture funding is working with The Jackson Laboratory to engineer mouse models that better recapitulate diseases potentially allowing Actio scientists to more accurately vet would be drugs
Sarepta has exclusive rights to the micro dystrophin gene therapy program initially developed at the Abigail Wexner Research Institute at Nationwide Childrens Hospital
In 2021 Verge formed a collaboration with Lilly to find and develop treatments for ALS or amyotrophic lateral sclerosis
For more information about ROCTAVIAN please visit BioMarincom or contact BioMarin Medical Information at medinfobmrncom
According to OrsoBio CEO, the investment from the pharmaceutical company reinforced a commitment to furthering new approaches They're quite interested in all four mechanisms of action but particularly interested in the protonophore program Subramanian said referring to one of its obesity drugs that targets the liver
The draw of epigenetic editing is the possibility of tweaking gene expression without fundamentally changing DNA. The idea is to turn a particular gene on or off to target a disease but avoid irreversible genetic changes that could end up harming a patient. Proponents say such therapies may even be able to tackle conditions that cant be helped by existing technologies
Since the introduction of VOXZOGO in 2021 the company has seen strong patient demand for the medicine worldwide
That medicine formerly known as buparlisib and now AN2025 is in latestage trials for a form of head and neck cancer and in earlier testing against other solid tumors
We are pleased to report strong dosedependent exonskipping and dystrophin expression results with monthly dosing of SRP5051  in ambulant and nonambulant patients
The passcode for the call is 2538387
Led by RA Capital Management the private placement financing is expected to close by Wednesday and raise about 150 million in gross proceeds for Taysha. The company held just over 45 million in cash at the end of June
In 2027 Moderna expects 8 billion to 15 billion in respiratory product sales
No matter which way you cut it while early the results on Roctavian thus far have been a clear disappointment wrote Stifel analyst Paul Matteis in a Nov 1 note to clients
Still despite the interest AI specialists havent been immune to the turbulence buffeting the biotech industry more broadly
Other drugmakers developing TYK2 inhibitors include Takeda  Priovant Therapeutics and Alumis
With the acquisition Alfasigma will gain Ocaliva a drug that treats the rare autoimmune disorder primary biliary cholangitis or PBC which had sales of 152 million in the first six months of the year
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Rosana Kapeller Permission granted by Rome Therapeutics In 2021 Rome brought in 77 million through its original Series B raise which Kapeller said was intended to bring its lead program through to clinical trials
Clinical studies of ROCTAVIAN did not include sufficient numbers of patients with HIV to determine whether the efficacy and safety differs compared to patients without HIV infection
And while more recent trials were successful, Zurzuvaes effects in these patients appeared to be modest overall and wane with time
Orum describes its therapy dubbed ORM6151 as an antibody enabled protein degrader merging the concept of an antibody drug conjugate with the field of protein degradation. Orum said it has received Food and Drug Administration clearance to enter Phase 1 testing in acute myeloid leukemia and myelodysplastic syndromes
The company is focused on developing small molecule drugs Artificial intelligence is at the heart of Superluminals approach which it said could help it advance new drug candidates more quickly. Biotechs that draw heavily on machine learning have attracted some nontraditional investors
Alnylam is headquartered in Cambridge MA
The ruling slashed Sages market value in half  as analysts cut their expectations for peak sales of the drug by as much as twothirds. The loss of the larger market also raised questions about whether Sages partner Biogen would stick with the medicine
AstraZeneca now has a stake in Verge as well
Because of the effect of reduced skeletal muscle mass on creatinine measurements creatinine may not be a reliable measure of kidney function in DMD patients
Instead studies found that Syfovre slows the growth of a marker of disease progression. That led some doctors to question its benefits when compared with the risk of side effects.
BioMarin is under no obligation and expressly disclaims any obligation to update or alter any forwardlooking statement whether as a result of new information future events or otherwise
ONPATTRO is also approved in the European Union Switzerland and Brazil for the treatment of hATTR amyloidosis in adults with Stage 1 or Stage 2 polyneuropathy and in Japan for the treatment of hATTR amyloidosis with polyneuropathy
The study tested a low and high dose of the therapy known as bemdaneprocel and found evidence across both groups that cells had engrafted and were surviving. Bluerock reported that the severity of disease had gotten better one year after treatment with patients in the highdose group showing the greatest improvement
The new promises for the future also come amid the backdrop of swiftly declining sales of COVID19 vaccines as people move on from the pandemic that made Moderna a household name
BrainStorm is now trying to take the most efficient path to get its therapy approved Lebovits added
But the company has also changed course multiple times and in 2021 pulled a drug application after Merck beat it to market with a similar medicine. It doesnt have any approved products and shares are currently worth less than 2 apiece
Kinnate joins the list of over 100 biotechs that have reduced their workforce after setbacks that could include failed drug trials or a lack of funding
After pulling off a biomedical triumph with its COVID19 vaccine Moderna on Wednesday put out a roadmap for investors that promises billions of dollars from new medicines
FDA concluded that the differences between Study 270201 Phase 12 and the Phase 3 study limited its ability to rely on the Phase 12 study to support durability of effect
Conference Call and Webcast Information Ascendis Pharma will host a conference call and webcast today at 830 am Eastern Time ET
Sages recent troubles are rooted in the Food and Drug Administration decision earlier this month to issue a narrow approval for a drug called Zurzuvae
Eli Lilly is looking to dethrone Novo with Mounjaro a dual acting injection that activates GLP1 and another target called GIP. Mounjaro has been approved in diabetes and could be launched soon for weight loss
GB0669, which targets the SARSCoV2 spike protein entered Phase 1 clinical testing over the summer. The experimental drug could be a more durable option for people who are immunocompromised and may have a harder time mounting a response to a COVID infection
Analysts expect a price similar to those for branded phosphate binders which have list prices around $1500 per month
The company's lead program is for a muscle disorder called facioscapulohumeral muscular dystrophy  or FSHD
Agenus has been working on cancer medicines for nearly three decades. The company was cofounded in 1994 and has been publicly traded since 2000. Over that time its churned through a variety of cancer drug approaches and established a lengthy list of partners among them Merck  Co Gilead Sciences and Bristol Myers Squibb
Massachusetts company had 80 employees and a handful of drug leads sourced from a machine learning platform that it boasted could speed up drug discovery timelines. Now with nearly 700 million in financing the company employs over 280 staff and plans to soon file an application for a second experimental drug to enter clinical trials in Europe said the CEO
Roche has invested heavily in Alzheimers drug development advancing two antibody therapies into Phase 3 testing only to find they didnt slow disease progression
FoundationOne CDx analyzes tissue samples for multiple biomarkers
Apellis finds faulty needles in probe of rare eye drug side effects The company doesnʼt know if the needles have caused retinal vasculitis in some people receiving its geographic atrophy drug Syfovre but is no longer recommending their use
Next up Moderna hopes is a vaccine for respiratory syncytial virus for which it expects to receive an FDA approval decision by next April
The rates are higher than those seen among the patients given intravenous Leqembi in the main part of the trial but Eisai notes how these side effects happen most often in the months just after patients begin taking antiamyloid therapies
Acelyrin lost more than half its market value Tuesday morning after the biotechnology companys lead drug failed in an closely watched Phase 2b3 trial
Alpha1 antitrypsin is crucial to protect the bodys tissues from being attacked by its own enzymes. When not produced or produced incorrectly it can lead to liver or lung disease
All statements other than statements of historical facts included in this press release regarding Ascendis future operations plans and objectives o f management are forward looking statements
Words such as believes anticipates plans expects will may intends prepares looks potential possible and similar expressions are intended to identify forwardlooking statements
In 2021 for example GSK mounted somewhat of a return to the field through a deal with Alector a Californiabased company attempting to use the immune system to fight neurodegeneration
Protein degraders have emerged as a competitive area of drug research over the last decade
In a conference call Monday company CFO said the rejection will not change Alnylams expectation that it will record 1213 billion in sales this year
Isotretinoin is not recommended in patients who are benefiting from ROCTAVIAN
These forwardlooking statements speak only as of the date hereof and we disclaim any obligation to update these statements except as may be required by law
The FDA also stated that this does not reflect their final regulatory decision on the Companys application
Results include All patients demonstrated robust transduction with mean microdystrophin expression of 554 of normal as measured by western blot
Japans Otsuka Pharmaceutical will work with Seattle startup Shape Therapeutics to develop gene therapies for the eye announcing Thursday a research collaboration centered on Shapes viral engineering technology
Financial Results On a GAAP basis for the three months ended June 30 2021 the Company reported a net loss of 814 million or 102 per basic and diluted share compared to a net loss of 1508 million reported for the same period of 2020 or 193 per basic and diluted share
Acelyrins setback now looms as two other clinicalstage biotech companies  Neumora and RayzeBio  ready their own IPOs
We hold leadership positions in Duchenne muscular dystrophy DMD and limbgirdle muscular dystrophies LGMDs and we currently have more than 40 programs in various stages of development
Flagship-backed Generate raises 273M as its first drugs move to the clinic Generateʼs Series C round appears to buck a trend of declining investment in platformbased biotechs this year
Leqembi is meant to slow the progression of Alzheimers by removing buildups of a sticky toxic protein known as amyloid beta
For more information about ONPATTRO including full Prescribing Information httpsctsbusinesswirecomctCT
About TransCon PTH TransCon PTH palopegteriparatide is an investigational prodrug in development for the treatment of adult patients with hypoparathyroidism
The documents released ahead of Wednesdays meeting show the FDA will make a strong case for why BrainStorms accumulated clinical trial evidence doesn't meet its bar for approval
View source version on businesswirecom  httpswwwbusinesswirecomnewshome20230316005693en Investor Contact Ian Estepan  6172744052 iestepansareptacom Media Contact Tracy Sorrentino  6173018566 tsorrentinosareptacom Source Sarepta Therapeutics Inc
Others have put off going public or looked to other opportunities like outlicensing or reverse mergers
This is a revolutionary approach with the potential to transform the care of patients with genetic and other diseases
Gain from sale of Priority Review Voucher In February 2021 the Company entered into an agreement to sell the rare pediatric disease Priority Review Voucher PRV it received from the FDA in connection with the approval of AMONDYS 45
Precision medicine approaches have led to tremendous successes in oncology and now we have the insights to be at the forefront of developing precision medicines to transform the treatment landscape for neuropsychiatry said Mitsuyuki Matsumoto Arialys scientific founder and chief scientific officer in a statement
Approximately 1 in 10000 people have Hemophilia A
Much like antibody drug conjugates emerged as a new and transformative treatment modality in certain cancers we see an opportunity for innovative radiopharmaceutical therapeutics to follow a similar path RayzeBio wrote in its regulatory filing
CRG022 is also being tested in patients who have not had other CART therapies as well as children with Bcell acute lymphoblastic leukemia
While the Danish biotechnology company has worked on vaccines since its founding in the early 90s it has largely relied on government contracts including for its smallpox and mpox shot Jynneos to stay afloat
Another from Prometheus Biosciences was bought by Merck  Co  while a third from Pfizer and Roivant Sciences has drawn industry interest
The company has so far released only limited details about its broader pipeline but is focused on autoimmune diseases cancer and neurodegeneration
According to Arialys data from a nonhuman primate disease model indicate the medicine could help treat ANRE or antiNMDA receptor encephalitis a rare potentially deadly condition in which autoantibodies impair proteins critical to brain function and memory
Targeted protein degradation is also an up-and-coming field putting Orum at the center of two fastmoving areas of research
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While investors have shown more appetite for biotechs in recent months the Aiolos financing stands out. The total funding tops any venture capital round for a biotech during the first quarter and comes close to the top five of the second quarter  according to the pharmaceutical industry information company Evaluate
UniQures stock price rose by as much as 14% on news of restructuring but remains about twothirds lower than where it traded earlier this summer before the company revealed mixed results from an early trial of its experimental Huntingtons disease treatment AMT130
The Company excludes collaboration revenue and transaction cost associated with the Roche transaction from its nonGAAP results
Generate Biomedicines on Thursday said it has raised another 273 million to fund its pipeline of drugs for autoimmune disorders cancers and infectious diseases
People with hypoparathyro idism need new treatment options and we are working with urgency to resolve the FDAs concerns  with the goal of bringing Trans Con PTH to patients in the US  As recently announced  145 of 154 clinical trial participants continue treatment with TransCon PTH for up to 3 years and the US
VOXZOGO is approved under accelerated approval based on an improvement in annualized growth velocity
The company will stop developing preclinical treatments for geographic atrophy and an unspecified brain disease as well as abandon a plan to study one of its marketed medicines alongside an RNAbased treatment
Courtesy of BioMarin Pharmaceutical BioMarin is bringing on a new chief executive as the gene therapy company struggles with an initially disappointing launch for an important new product
There are no data on the use of ROCTAVIAN in pregnant women or regarding lactation
We also advanced our broad genetic medicine portfolio
After Idorsia reported positive Phase 3 results in May 2022 analysts at Jeffries predicted it could eventually bring in sales of 25 billion a year in the US and Europe
To be built at Technical University of Denmark in Lyngby the facility will be available for use by private companies as well as academic researchers. Its main function will be to help the final development steps and upscaling of production to initiate clinical trials. Construction is slated to begin in 2024 with the facility expected to be operational in 2027
In Verves case, its treatment changes an A in the sequence for a gene called PCSK9 to a G inactivating the gene
Initially CRISPR was often described as molecular scissors that could cut through DNA and make changes to alter the course of a disease
Isotretinoin Efavirenz and HIV Positive Patients
Beam to lay off 100 employees seek partners in research restructuring The base editing specialist is prioritizing medicines for sickle cell and certain other diseases while narrowing or trimming other research
Though a total dissolution is unusual in the biotechnology industry its not unheard of. Genocea Biosciences for example found itself in a similar position last year
Importantly these early data indicate that the miRNAResponsive AutoRegulatory Element miRARE technology is mediating MECP2 expression in the CNS on a cellbycell basis supporting the regulatory control of miRARE
For TSHA102 in Rett syndrome we believe the initial safety profile and significant clinical improvements seen in the first adult patient with severe disease four weeks posttreatment reinforces the transformative potential of our gene therapy to address the root cause of Rett syndrome
Agenus to lay off 25 of staff trim pipeline in cancer drug push The biotech has joined a list of more than 100 companies to cut jobs this year according to BioPharma Dive data
Seagen partners with Nurix to cr eate new kind of cancer drug. In a multiyear research alliance the companies aim to combine Seagenʼs antibody technology with compounds that can degrade cancerdriving proteins
But to make sure theres enough room for these new cells patients first undergo myeloablation  a chemotherapy regimen that can be very difficult on their bodies and comes with the risk of infertility. Older patients may not be healthy enough to receive this treatment