Bio­gen bags an AAV gene ther­a­py pro­gram from Mass­a­chu­setts Eye and Ear; Biotechs raised $1B-plus in lat­est round of fol­low-ons

→ Bio­gen has picked up a new, pre­clin­i­cal gene ther­a­py pro­gram from Mass­a­chu­setts Eye and Ear for in­her­it­ed reti­nal de­gen­er­a­tion due to mu­ta­tions in the PRPF31 gene, among the most com­mon caus­es for au­to­so­mal dom­i­nant re­tini­tis pig­men­tosa. They’re build­ing on the work of Har­vard’s Er­ic Pierce. “The treat­ment of IRDs with high­ly ef­fec­tive AAV-based gene ther­a­pies is core to Bio­gen’s oph­thal­mol­o­gy strat­e­gy,” said Chris Hen­der­son, the re­search head at Bio­gen. “This agree­ment un­der­scores our com­mit­ment to that strat­e­gy and builds off of our ac­qui­si­tion of Night­star Ther­a­peu­tics in 2019 and our ac­tive clin­i­cal tri­als of gene ther­a­pies for dif­fer­ent ge­net­ic forms of IRD.”

→ Sarep­ta has inked a col­lab­o­ra­tion with Hansa to de­vel­op their ex­per­i­men­tal drug im­li­fi­dase as a pre-treat­ment for their gene ther­a­pies. The drug is in­tend­ed for use in pa­tients who have neu­tral­iz­ing an­ti­bod­ies that would pre­vent gene ther­a­pies for Duchenne mus­cu­lar dy­s­tro­phy and Limb-gir­dle mus­cu­lar dy­s­tro­phy from work­ing. Hansa gets a $10 mil­lion up­front and up to $397.5 mil­lion in mile­stones.

It’s been rain­ing mon­ey on Wall Street — at least when it comes to drug de­vel­op­ers. CRISPR Ther­a­peu­tics $CR­SP and Ac­celeron each raised a whop­ping $450 mil­lion this week af­ter pric­ing fol­low-on of­fer­ings. CRISPR priced 6,428,572 com­mon shares at a pub­lic of­fer­ing price of $70.00 per share, while Ac­celeron $XL­RN auc­tioned off 4,864,864 shares of com­mon stock at a price to the pub­lic of $92.50 per share.

The transat­lantic biotech play­er Immat­ics has com­plet­ed its flip on­to Nas­daq through the Arya Sci­ences Ac­qui­si­tion Corp. The can­cer drug biotech will trade as $IMTX af­ter it raised $253 mil­lion in the process. The SPAC was set up by Per­cep­tive Ad­vi­sors.

Seat­tle-based Ne­oleukin Ther­a­peu­tics, mean­while, raised $76.2 mil­lion $NLTX for its work on pro­tein ther­a­peu­tics.

At the In­flec­tion Point for the Next Gen­er­a­tion of Can­cer Im­munother­a­py

While oncology researchers have long pursued the potential of cellular immunotherapies for the treatment of cancer, it was unclear whether these therapies would ever reach patients due to the complexity of manufacturing and costs of development. Fortunately, the recent successful development and regulatory approval of chimeric antigen receptor-engineered T (CAR-T) cells have demonstrated the significant benefit of these therapies to patients.

All about Omi­cron; We need more Covid an­tivi­rals; GSK snags Pfiz­er’s vac­cine ex­ec; Janet Wood­cock’s fu­ture at FDA; and more

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Merck's new antiviral molnupiravir (Quality Stock Arts / Shutterstock)

As Omi­cron spread looms, oral an­tivi­rals ap­pear to be one of the best de­fens­es — now we just need more

After South African scientists reported a new Covid-19 variant — dubbed Omicron by the WHO — scientists became concerned about how effective vaccines and monoclonal antibodies might be against it, which has more than 30 mutations in the spike protein.

“I think it is super worrisome,” Dartmouth professor and Adagio co-founder and CEO Tillman Gerngross told Endpoints News this weekend. Moderna CEO Stéphane Bancel echoed similar concerns, telling the Financial Times that experts warned him, “This is not going to be good.”

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Biospec­i­men M&A: Dis­cov­ery ac­quires Al­bert Li's he­pa­to­cyte project; PhI­II tri­al on Bay­er's Nube­qa reached pri­ma­ry end­point

Discovery Life Sciences has acquired what claims to be the Maryland-based host of the world’s largest hepatocyte inventory, known as IVAL, to help researchers select more effective and safer drug candidates in the future.

The combined companies will now serve a wider range of drug research and development scientists, according to Albert Li, who founded IVAL in 2004 and is set to join the Discovery leadership team as the CSO of pharmacology and toxicology.

Lisa Deschamps, AviadoBio CEO

Ex-No­var­tis busi­ness head hops over to a gene ther­a­py start­up — and she's reeled in $80M for a dash to the clin­ic

Neurologist and King’s College London professor Christopher Shaw has been researching neurodegenerative diseases like ALS and collaborating with drugmakers for the last 25 years in the hopes of pushing new therapies forward. But unfortunately, none of those efforts have come anywhere close to fruition.

“So, you know, after 20 years in the game, I said, ‘Let’s try and do it ourselves,’” he told Endpoints News. 

In­cor­po­rat­ing Ex­ter­nal Da­ta in­to Clin­i­cal Tri­als: Com­par­ing Dig­i­tal Twins to Ex­ter­nal Con­trol Arms

Most drug development professionals are familiar with the nerve-racking wait for the read-out of a large trial. If it’s negative, is the investigational therapy ineffective? Or could the failure result from an unforeseen flaw in the design or execution of the protocol, rather than a lack of efficacy? The team could spend weeks analyzing data, but a definitive answer may be elusive due to insufficient power for such analyses in the already completed trial. These problems are only made worse if the trial had lower enrollment, or higher dropout than expected due to an unanticipated event like COVID-19. And if a trial is negative, the next one is likely to be larger and more costly — if it happens at all.

Ab­b­Vie tacks on a new warn­ing to Rin­voq la­bel as safe­ty frets crimp JAK class

The safety problems that continue to plague the JAK class as new data highlight some severe side effects are casting a large shadow over AbbVie’s Rinvoq.

As a result of a recent readout highlighting major adverse cardiac events (MACE), malignancy, mortality and thrombosis with Xeljanz a couple of months ago, AbbVie put out a notice late Friday afternoon that it is adding the new class risks to its label for their rival drug.

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Pfiz­er, Am­gen and Janssen seek fur­ther clar­i­ty on FDA's new ben­e­fit-risk guid­ance

Three top biopharma companies are seeking more details from the FDA on how the agency conducts its benefit-risk assessments for new drugs and biologics.

While Pfizer, Amgen and Janssen praised the agency for further spelling out its thinking on the subject in a new draft guidance, including a discussion of patient experience data as part of the assessment, the companies said the FDA could’ve included more specifics in the 20-page draft document.

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Re­searchers move clos­er to de­ci­pher­ing blood clots from As­traZeneca, J&J's Covid-19 vac­cines

Researchers may be nearing an answer for the mysterious and life-threatening blood clots that appeared on very rare occasions in people who received the J&J or AstraZeneca Covid-19 vaccine.

The new work builds on an early hypothesis researchers in Norway put forward last spring, when the cases first cropped up. They proposed the events were similar to blood clots that can occur in a small subset of patients who receive heparin, one of the most commonly used blood thinners.